Jerry R Mendell

Contact Information

Call us at: (614) 722-4877

Fax us at: (614) 355-7686

Pediatrics700 Children’s DrColumbus, OH 43205 (map)

Learn more about Jerry R. Mendell

Biography

Jerry R. Mendell, MD, is an attending neurologist at Nationwide Children’s Hospital, the Dwight E. Peters and Juanita R. Curran Endowed Chair in Pediatric Research at the Abigail Wexner Research Institute at Nationwide Children’s Hospital and professor of Pediatrics and Neurology at Nationwide Children’s and The Ohio State University. Dr. Mendell joined the Research Institute at Nationwide Children’s fulltime in 2004, recruited by Philip Johnson, the Head of the Center for Gene Therapy at that time. Shortly after, leadership of the Center changed and provided the opportunity to build and establish a program in Clinical Translation with emphasis on neuromuscular disease. At Nationwide Children’s, he holds academic positions as Professor of Pediatrics and Neurology and holds the Curran-Peters Chair of Pediatric Research. He is a clinician scientist with decades of experience. A career emphasis began with training in neuromuscular disease at the National Institutes of Health, directing laboratory projects that could be carried to the bedside. He was recruited to Columbus to direct the Neuromuscular Center at Ohio State. His life’s work has emphasized clinical translation. He has published more than 370 articles with a focus on neuromuscular disease and authored books on muscle disease, peripheral nerve disorders, and most recently, gene therapy for muscle disease. He was the principal investigator on the prednisone clinical trial in DMD in 1989 that has profoundly influenced clinical care. He was the PI on the exon skipping trial using eteplirsen, now FDA approved for clinical use in 2016. He was the PI on the first gene therapy trials for DMD and LGMD and was the PI on the SMA gene therapy trial that was FDA approved for systemic delivery for infants with SMA type 1 in 2017. The results of the first systemic gene therapy trial for DMD is now moving to Phase 3. Results from the Phase 1/2 micro-dystrophin trial will be published in June 2020.

Patient Care

Locations

Westerville Surgery Center

Main Campus of Nationwide Children’s Hospital

Academic and Clinical Areas

Neuromuscular Disorders

Physician Team

Neurology

Physician Team

Center for Gene Therapy

Principal Investigator

Neurology Residency

Faculty

Primary Department

Pediatrics

Primary Section

Neurology

Awards, Honors & Organizations

Juanita Curran and Dwight Peters Endowed Chair, 2010 Distinguished Scholar Award, The Ohio State University, Presented by the President, 2009 S. Mouchly Small Scientific Achievement Award, The Muscular Dystrophy Association for Significant Contributions to Neuromuscular Disease Research, 2004 Listed, Best Doctors in America

Research

Lab(s)

Center for Gene Therapy

The Mendell Lab is devoted to the development of potential treatments for neuromuscular diseases including Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophy (LGMD). DMD causes progressive muscle weakness that usually confines patients to a wheelchair in their early teens. Limb-girdle muscular dystrophies are a group of at least 13 diseases classified by their disease causing genes. It causes loss of muscle mass and strength in voluntary muscles, mainly those around the hips and shoulders. These diseases are caused by genetic defects that lead to missing or non-functional proteins. This makes gene replacement or gene therapy a good treatment option for these diseases. Our major research goal is successful gene therapy, by replacing genes for DMD and LGMD using adeno-associated virus (AAV) vectors to carry a copy of the corrected gene to the muscle cells. In some instances such as the dystrophin gene (the largest gene identified) “mini” and “micro” genes must be used. This can be done by direct injection into muscle (IM) or by way of the blood stream (vascular delivery). Muscular dystrophy can also be treated with follistatin by way of IM injection with adeno-associated virus (AAV) vectors to carry the follistatin gene in IBM and BMD. It inhibits myostatin, promoting muscle growth and preventing deterioration of existing muscle. Another therapeutic approach is exon skipping, treating primarily DMD, using synthetic molecules that induce skipping or read-through of the damaged portion of the dystrophin gene. This is a mutation-specific treatment with each molecule designed to skip a certain exon(s). This leads to restoration of the reading frame and production of an internally truncated partially functional dystrophin protein.In addition to developing potential treatments for neuromuscular diseases, the Mendell laboratory also concentrates on the diagnosis and characterization of neuromusclar diseases. The Mendell lab also serves as the diagnostic lab of muscle biopsies for the Anatomic Pathology Department of Nationwide Children’s Hospital. We are currently conducting a newborn screening study for DMD with The Research Institute at Nationwide Children’s Hospital and Cincinnati Children’s Hospital to identify possible cases in young males in the state of Ohio. The lab also has the resources of the United Dystrophinopathy Project (UDP) available for genetic screening and characterization of dystrophinopathies that cause DMD. The UDP directly sequences the coding and regulatory regions of the dystrophin gene, leading to faster and more specific characterization of mutations of many dystrophinopathy patients. The Mendell lab also has resources here at Nationwide Children’s Hospital to identify and sequence other genes that cause muscular dystrophies. Publications

                  Mitelman O, Abdel-Hamid HZ, Byrne BJ, Connolly AM, Heydemann P, Proud C, Shieh PB, Wagner KR, Dugar A, Santra S, Signorovitch J, Goemans N, investigators from the LNMRC Natural History study., McDonald CM, investigators from the CINRG Duchenne National History Study., Mercuri E, investigators from The DMD Italian Group., Mendell JR. A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirsen Treatment. J Neuromuscul Dis. 2022; 9: 39-52.

                


                  Day JW, Mendell JR, Mercuri E, Finkel RS, Strauss KA, Kleyn A, Tauscher-Wisniewski S, Tukov FF, Reyna SP, Chand DH. Correction to: Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy. Drug Saf. 2021 Dec 23;  

                


                  Sahenk Z, Ozes B, Murrey D, Myers M, Moss K, Yalvac ME, Ridgley A, Chen L, Mendell JR. Systemic delivery of AAVrh74.tMCK.hCAPN3 rescues the phenotype in a mouse model for LGMD2A/R1. Mol Ther Methods Clin Dev. 2021 Sep 10; 22: 401-414.

                


                  Al-Zaidy SA, Kolb SJ, Lowes L, Alfano LN, Shell R, Church KR, Nagendran S, Sproule DM, Feltner DE, Wells C, Ogrinc F, Menier M, L'Italien J, Arnold WD, Kissel JT, Kaspar BK, Mendell JR. AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort. J Neuromuscul Dis. 2019 Jul 30;  

                


                  Connolly AM, Zaidman CM, Golumbek PT, Cradock MM, Flanigan KM, Kuntz NL, Finkel RS, McDonald CM, Iannaccone ST, Anand P, Siener CA, Florence JM, Lowes LP, Alfano LN, Johnson LB, Nicorici A, Nelson LL, Mendell JR, MDA DMD Clinical Research Network.. Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy. Muscle Nerve. 2019 Jun; 59: 650-657.

                


                  Ke Q, Zhao ZY, Mendell JR, Baker M, Wiley V, Kwon JM, Alfano LN, Connolly AM, Jay C, Polari H, Ciafaloni E, Qi M, Griggs RC, Gatheridge MA. Progress in treatment and newborn screening for Duchenne muscular dystrophy and spinal muscular atrophy. World J Pediatr. 2019 Jun; 15: 219-225.

                


                  Al-Zaidy SA, Mendell JR. From Clinical Trials to Clinical Practice: Practical Considerations for Gene Replacement Therapy in SMA Type 1. Pediatr Neurol. 2019 Jun 13;  

                


                  Alfano LN, Charleston JS, Connolly AM, Cripe L, Donoghue C, Dracker R, Dworzak J, Eliopoulos H, Frank DE, Lewis S, Lucas K, Lynch J, Milici AJ, Flynt A, Naughton E, Rodino-Klapac LR, Sahenk Z, Schnell FJ, Young GD, Mendell JR, Lowes LP. Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy. Medicine (Baltimore). 2019 Jun; 98: e15858.

                


                  Lowes LP, Alfano LN, Arnold WD, Shell R, Prior TW, McColly M, Lehman KJ, Church K, Sproule DM, Nagendran S, Menier M, Feltner DE, Wells C, Kissel JT, Al-Zaidy S, Mendell J. Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy. Pediatr Neurol. 2019 May 13;  

                


                  Mendell JR, Chicoine LG, Al-Zaidy SA, Sahenk Z, Lehman K, Lowes L, Miller N, Alfano L, Galliers B, Lewis S, Murrey D, Peterson E, Griffin DA, Church K, Cheatham S, Cheatham J, Hogan MJ, Rodino-Klapac LR. Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion. Hum Gene Ther. 2019 Apr 19;  

                


                  Al-Zaidy S, Pickard AS, Kotha K, Alfano LN, Lowes L, Paul G, Church K, Lehman K, Sproule DM, Dabbous O, Maru B, Berry K, Arnold WD, Kissel JT, Mendell JR, Shell R. Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy. Pediatr Pulmonol. 2019 Feb; 54: 179-185.

                


                  Khan N, Eliopoulos H, Han L, Kinane TB, Lowes LP, Mendell JR, Gordish-Dressman H, Henricson EK, McDonald CM, Eteplirsen Investigators and the CINRG DNHS Investigators.. Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy. J Neuromuscul Dis. 2019; 6: 213-225.

                


                  Diaz-Manera J, Fernandez-Torron R, LLauger J, James MK, Mayhew A, Smith FE, Moore UR, Blamire AM, Carlier PG, Rufibach L, Mittal P, Eagle M, Jacobs M, Hodgson T, Wallace D, Ward L, Smith M, Stramare R, Rampado A, Sato N, Tamaru T, Harwick B, Rico Gala S, Turk S, Coppenrath EM, Foster G, Bendahan D, Le Fur Y, Fricke ST, Otero H, Foster SL, Peduto A, Sawyer AM, Hilsden H, Lochmuller H, Grieben U, Spuler S, Tesi Rocha C, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Harms M, Pestronk A, Krause S, Schreiber-Katz O, Walter MC, Paradas C, Hogrel JY, Stojkovic T, Takeda S, Mori-Yoshimura M, Bravver E, Sparks S, Bello L, Semplicini C, Pegoraro E, Mendell JR, Bushby K, Straub V, Jain COS Consortium.. Muscle MRI in patients with dysferlinopathy: pattern recognition and implications for clinical trials. J Neurol Neurosurg Psychiatry. 2018 Oct; 89: 1071-1081.

                


                  Mendell JR. Therapy for Spinal Muscular Atrophy. N Engl J Med. 2018 Feb 1; 378: 487.

                


                  Moore UR, Jacobs M, Fernandez-Torron R, Jang J, James MK, Mayhew A, Rufibach L, Mittal P, Eagle M, Cnaan A, Carlier PG, Blamire A, Hilsden H, Lochmüller H, Grieben U, Spuler S, Tesi Rocha C, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Harms M, Pestronk A, Krause S, Schreiber-Katz O, Walter MC, Paradas C, Hogrel JY, Stojkovic T, Takeda S, Mori-Yoshimura M, Bravver E, Sparks S, Diaz-Manera J, Bello L, Semplicini C, Pegoraro E, Mendell JR, Bushby K, Straub V. Teenage exercise is associated with earlier symptom onset in dysferlinopathy: a retrospective cohort study. J Neurol Neurosurg Psychiatry. 2018 Jan 29;  

                


                  Willcocks RJ, Triplett WT, Lott DJ, Forbes SC, Batra A, Sweeney HL, Mendell JR, Vandenborne K, Walter GA. Leg muscle MRI in identical twin boys with duchenne muscular dystrophy. Muscle Nerve. 2018 Jan 24;  

View More Publications

Education

Date of Appointment at Nationwide Children’s Hospital: 08/27/2004

Board Certifications

Neurology

Fellowship

National Institutes of Health

Date Completed: 07/31/1971

Residency

National Institutes of Health

Date Completed: 06/30/1970

Residency

Columbia Presbyterian Medical Center - New York

Date Completed: 06/30/1969

Medical School

University of Texas Southwestern Medical Center

Date Completed: 06/30/1966

Professional Experience

1992 - 2004 Dept of Neurology, The Ohio State University, Chair

Contact Information

Pediatrics

Call us at: (614) 722-4877

Fax us at: (614) 355-7686

                    700 Children's DrColumbus, OH 43205 (map)

Contact Information

Call us at: (614) 722-4877

Fax us at: (614) 355-7686

Pediatrics700 Children’s DrColumbus, OH 43205 (map)

Learn more about Jerry R. Mendell

Biography

Jerry R. Mendell, MD, is an attending neurologist at Nationwide Children’s Hospital, the Dwight E. Peters and Juanita R. Curran Endowed Chair in Pediatric Research at the Abigail Wexner Research Institute at Nationwide Children’s Hospital and professor of Pediatrics and Neurology at Nationwide Children’s and The Ohio State University. Dr. Mendell joined the Research Institute at Nationwide Children’s fulltime in 2004, recruited by Philip Johnson, the Head of the Center for Gene Therapy at that time. Shortly after, leadership of the Center changed and provided the opportunity to build and establish a program in Clinical Translation with emphasis on neuromuscular disease. At Nationwide Children’s, he holds academic positions as Professor of Pediatrics and Neurology and holds the Curran-Peters Chair of Pediatric Research. He is a clinician scientist with decades of experience. A career emphasis began with training in neuromuscular disease at the National Institutes of Health, directing laboratory projects that could be carried to the bedside. He was recruited to Columbus to direct the Neuromuscular Center at Ohio State. His life’s work has emphasized clinical translation. He has published more than 370 articles with a focus on neuromuscular disease and authored books on muscle disease, peripheral nerve disorders, and most recently, gene therapy for muscle disease. He was the principal investigator on the prednisone clinical trial in DMD in 1989 that has profoundly influenced clinical care. He was the PI on the exon skipping trial using eteplirsen, now FDA approved for clinical use in 2016. He was the PI on the first gene therapy trials for DMD and LGMD and was the PI on the SMA gene therapy trial that was FDA approved for systemic delivery for infants with SMA type 1 in 2017. The results of the first systemic gene therapy trial for DMD is now moving to Phase 3. Results from the Phase 1/2 micro-dystrophin trial will be published in June 2020.

Patient Care

Locations

Westerville Surgery Center

Main Campus of Nationwide Children’s Hospital

Academic and Clinical Areas

Neuromuscular Disorders

Physician Team

Neurology

Physician Team

Center for Gene Therapy

Principal Investigator

Neurology Residency

Faculty

Primary Department

Pediatrics

Primary Section

Neurology

Awards, Honors & Organizations

Juanita Curran and Dwight Peters Endowed Chair, 2010 Distinguished Scholar Award, The Ohio State University, Presented by the President, 2009 S. Mouchly Small Scientific Achievement Award, The Muscular Dystrophy Association for Significant Contributions to Neuromuscular Disease Research, 2004 Listed, Best Doctors in America

Research

Lab(s)

Center for Gene Therapy

The Mendell Lab is devoted to the development of potential treatments for neuromuscular diseases including Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophy (LGMD). DMD causes progressive muscle weakness that usually confines patients to a wheelchair in their early teens. Limb-girdle muscular dystrophies are a group of at least 13 diseases classified by their disease causing genes. It causes loss of muscle mass and strength in voluntary muscles, mainly those around the hips and shoulders. These diseases are caused by genetic defects that lead to missing or non-functional proteins. This makes gene replacement or gene therapy a good treatment option for these diseases. Our major research goal is successful gene therapy, by replacing genes for DMD and LGMD using adeno-associated virus (AAV) vectors to carry a copy of the corrected gene to the muscle cells. In some instances such as the dystrophin gene (the largest gene identified) “mini” and “micro” genes must be used. This can be done by direct injection into muscle (IM) or by way of the blood stream (vascular delivery). Muscular dystrophy can also be treated with follistatin by way of IM injection with adeno-associated virus (AAV) vectors to carry the follistatin gene in IBM and BMD. It inhibits myostatin, promoting muscle growth and preventing deterioration of existing muscle. Another therapeutic approach is exon skipping, treating primarily DMD, using synthetic molecules that induce skipping or read-through of the damaged portion of the dystrophin gene. This is a mutation-specific treatment with each molecule designed to skip a certain exon(s). This leads to restoration of the reading frame and production of an internally truncated partially functional dystrophin protein.In addition to developing potential treatments for neuromuscular diseases, the Mendell laboratory also concentrates on the diagnosis and characterization of neuromusclar diseases. The Mendell lab also serves as the diagnostic lab of muscle biopsies for the Anatomic Pathology Department of Nationwide Children’s Hospital. We are currently conducting a newborn screening study for DMD with The Research Institute at Nationwide Children’s Hospital and Cincinnati Children’s Hospital to identify possible cases in young males in the state of Ohio. The lab also has the resources of the United Dystrophinopathy Project (UDP) available for genetic screening and characterization of dystrophinopathies that cause DMD. The UDP directly sequences the coding and regulatory regions of the dystrophin gene, leading to faster and more specific characterization of mutations of many dystrophinopathy patients. The Mendell lab also has resources here at Nationwide Children’s Hospital to identify and sequence other genes that cause muscular dystrophies. Publications

                  Mitelman O, Abdel-Hamid HZ, Byrne BJ, Connolly AM, Heydemann P, Proud C, Shieh PB, Wagner KR, Dugar A, Santra S, Signorovitch J, Goemans N, investigators from the LNMRC Natural History study., McDonald CM, investigators from the CINRG Duchenne National History Study., Mercuri E, investigators from The DMD Italian Group., Mendell JR. A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirsen Treatment. J Neuromuscul Dis. 2022; 9: 39-52.

                


                  Day JW, Mendell JR, Mercuri E, Finkel RS, Strauss KA, Kleyn A, Tauscher-Wisniewski S, Tukov FF, Reyna SP, Chand DH. Correction to: Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy. Drug Saf. 2021 Dec 23;  

                


                  Sahenk Z, Ozes B, Murrey D, Myers M, Moss K, Yalvac ME, Ridgley A, Chen L, Mendell JR. Systemic delivery of AAVrh74.tMCK.hCAPN3 rescues the phenotype in a mouse model for LGMD2A/R1. Mol Ther Methods Clin Dev. 2021 Sep 10; 22: 401-414.

                


                  Al-Zaidy SA, Kolb SJ, Lowes L, Alfano LN, Shell R, Church KR, Nagendran S, Sproule DM, Feltner DE, Wells C, Ogrinc F, Menier M, L'Italien J, Arnold WD, Kissel JT, Kaspar BK, Mendell JR. AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort. J Neuromuscul Dis. 2019 Jul 30;  

                


                  Connolly AM, Zaidman CM, Golumbek PT, Cradock MM, Flanigan KM, Kuntz NL, Finkel RS, McDonald CM, Iannaccone ST, Anand P, Siener CA, Florence JM, Lowes LP, Alfano LN, Johnson LB, Nicorici A, Nelson LL, Mendell JR, MDA DMD Clinical Research Network.. Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy. Muscle Nerve. 2019 Jun; 59: 650-657.

                


                  Ke Q, Zhao ZY, Mendell JR, Baker M, Wiley V, Kwon JM, Alfano LN, Connolly AM, Jay C, Polari H, Ciafaloni E, Qi M, Griggs RC, Gatheridge MA. Progress in treatment and newborn screening for Duchenne muscular dystrophy and spinal muscular atrophy. World J Pediatr. 2019 Jun; 15: 219-225.

                


                  Al-Zaidy SA, Mendell JR. From Clinical Trials to Clinical Practice: Practical Considerations for Gene Replacement Therapy in SMA Type 1. Pediatr Neurol. 2019 Jun 13;  

                


                  Alfano LN, Charleston JS, Connolly AM, Cripe L, Donoghue C, Dracker R, Dworzak J, Eliopoulos H, Frank DE, Lewis S, Lucas K, Lynch J, Milici AJ, Flynt A, Naughton E, Rodino-Klapac LR, Sahenk Z, Schnell FJ, Young GD, Mendell JR, Lowes LP. Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy. Medicine (Baltimore). 2019 Jun; 98: e15858.

                


                  Lowes LP, Alfano LN, Arnold WD, Shell R, Prior TW, McColly M, Lehman KJ, Church K, Sproule DM, Nagendran S, Menier M, Feltner DE, Wells C, Kissel JT, Al-Zaidy S, Mendell J. Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy. Pediatr Neurol. 2019 May 13;  

                


                  Mendell JR, Chicoine LG, Al-Zaidy SA, Sahenk Z, Lehman K, Lowes L, Miller N, Alfano L, Galliers B, Lewis S, Murrey D, Peterson E, Griffin DA, Church K, Cheatham S, Cheatham J, Hogan MJ, Rodino-Klapac LR. Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion. Hum Gene Ther. 2019 Apr 19;  

                


                  Al-Zaidy S, Pickard AS, Kotha K, Alfano LN, Lowes L, Paul G, Church K, Lehman K, Sproule DM, Dabbous O, Maru B, Berry K, Arnold WD, Kissel JT, Mendell JR, Shell R. Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy. Pediatr Pulmonol. 2019 Feb; 54: 179-185.

                


                  Khan N, Eliopoulos H, Han L, Kinane TB, Lowes LP, Mendell JR, Gordish-Dressman H, Henricson EK, McDonald CM, Eteplirsen Investigators and the CINRG DNHS Investigators.. Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy. J Neuromuscul Dis. 2019; 6: 213-225.

                


                  Diaz-Manera J, Fernandez-Torron R, LLauger J, James MK, Mayhew A, Smith FE, Moore UR, Blamire AM, Carlier PG, Rufibach L, Mittal P, Eagle M, Jacobs M, Hodgson T, Wallace D, Ward L, Smith M, Stramare R, Rampado A, Sato N, Tamaru T, Harwick B, Rico Gala S, Turk S, Coppenrath EM, Foster G, Bendahan D, Le Fur Y, Fricke ST, Otero H, Foster SL, Peduto A, Sawyer AM, Hilsden H, Lochmuller H, Grieben U, Spuler S, Tesi Rocha C, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Harms M, Pestronk A, Krause S, Schreiber-Katz O, Walter MC, Paradas C, Hogrel JY, Stojkovic T, Takeda S, Mori-Yoshimura M, Bravver E, Sparks S, Bello L, Semplicini C, Pegoraro E, Mendell JR, Bushby K, Straub V, Jain COS Consortium.. Muscle MRI in patients with dysferlinopathy: pattern recognition and implications for clinical trials. J Neurol Neurosurg Psychiatry. 2018 Oct; 89: 1071-1081.

                


                  Mendell JR. Therapy for Spinal Muscular Atrophy. N Engl J Med. 2018 Feb 1; 378: 487.

                


                  Moore UR, Jacobs M, Fernandez-Torron R, Jang J, James MK, Mayhew A, Rufibach L, Mittal P, Eagle M, Cnaan A, Carlier PG, Blamire A, Hilsden H, Lochmüller H, Grieben U, Spuler S, Tesi Rocha C, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Harms M, Pestronk A, Krause S, Schreiber-Katz O, Walter MC, Paradas C, Hogrel JY, Stojkovic T, Takeda S, Mori-Yoshimura M, Bravver E, Sparks S, Diaz-Manera J, Bello L, Semplicini C, Pegoraro E, Mendell JR, Bushby K, Straub V. Teenage exercise is associated with earlier symptom onset in dysferlinopathy: a retrospective cohort study. J Neurol Neurosurg Psychiatry. 2018 Jan 29;  

                


                  Willcocks RJ, Triplett WT, Lott DJ, Forbes SC, Batra A, Sweeney HL, Mendell JR, Vandenborne K, Walter GA. Leg muscle MRI in identical twin boys with duchenne muscular dystrophy. Muscle Nerve. 2018 Jan 24;  

View More Publications

Education

Date of Appointment at Nationwide Children’s Hospital: 08/27/2004

Board Certifications

Neurology

Fellowship

National Institutes of Health

Date Completed: 07/31/1971

Residency

National Institutes of Health

Date Completed: 06/30/1970

Residency

Columbia Presbyterian Medical Center - New York

Date Completed: 06/30/1969

Medical School

University of Texas Southwestern Medical Center

Date Completed: 06/30/1966

Professional Experience

1992 - 2004 Dept of Neurology, The Ohio State University, Chair

Contact Information

Pediatrics

Call us at: (614) 722-4877

Fax us at: (614) 355-7686

                    700 Children's DrColumbus, OH 43205 (map)

Contact Information

Call us at: (614) 722-4877

Fax us at: (614) 355-7686

Pediatrics700 Children’s DrColumbus, OH 43205 (map)

Learn more about Jerry R. Mendell

Contact Information

• Call us at:
• (614) 722-4877
• Fax us at:
• (614) 355-7686
• Pediatrics700 Children’s DrColumbus, OH 43205 (map)

Learn more about Jerry R. Mendell

Biography

Jerry R. Mendell, MD, is an attending neurologist at Nationwide Children’s Hospital, the Dwight E. Peters and Juanita R. Curran Endowed Chair in Pediatric Research at the Abigail Wexner Research Institute at Nationwide Children’s Hospital and professor of Pediatrics and Neurology at Nationwide Children’s and The Ohio State University. Dr. Mendell joined the Research Institute at Nationwide Children’s fulltime in 2004, recruited by Philip Johnson, the Head of the Center for Gene Therapy at that time. Shortly after, leadership of the Center changed and provided the opportunity to build and establish a program in Clinical Translation with emphasis on neuromuscular disease. At Nationwide Children’s, he holds academic positions as Professor of Pediatrics and Neurology and holds the Curran-Peters Chair of Pediatric Research. He is a clinician scientist with decades of experience. A career emphasis began with training in neuromuscular disease at the National Institutes of Health, directing laboratory projects that could be carried to the bedside. He was recruited to Columbus to direct the Neuromuscular Center at Ohio State. His life’s work has emphasized clinical translation. He has published more than 370 articles with a focus on neuromuscular disease and authored books on muscle disease, peripheral nerve disorders, and most recently, gene therapy for muscle disease. He was the principal investigator on the prednisone clinical trial in DMD in 1989 that has profoundly influenced clinical care. He was the PI on the exon skipping trial using eteplirsen, now FDA approved for clinical use in 2016. He was the PI on the first gene therapy trials for DMD and LGMD and was the PI on the SMA gene therapy trial that was FDA approved for systemic delivery for infants with SMA type 1 in 2017. The results of the first systemic gene therapy trial for DMD is now moving to Phase 3. Results from the Phase 1/2 micro-dystrophin trial will be published in June 2020.

Biography

Jerry R. Mendell, MD, is an attending neurologist at Nationwide Children’s Hospital, the Dwight E. Peters and Juanita R. Curran Endowed Chair in Pediatric Research at the Abigail Wexner Research Institute at Nationwide Children’s Hospital and professor of Pediatrics and Neurology at Nationwide Children’s and The Ohio State University. Dr. Mendell joined the Research Institute at Nationwide Children’s fulltime in 2004, recruited by Philip Johnson, the Head of the Center for Gene Therapy at that time. Shortly after, leadership of the Center changed and provided the opportunity to build and establish a program in Clinical Translation with emphasis on neuromuscular disease. At Nationwide Children’s, he holds academic positions as Professor of Pediatrics and Neurology and holds the Curran-Peters Chair of Pediatric Research. He is a clinician scientist with decades of experience. A career emphasis began with training in neuromuscular disease at the National Institutes of Health, directing laboratory projects that could be carried to the bedside. He was recruited to Columbus to direct the Neuromuscular Center at Ohio State. His life’s work has emphasized clinical translation. He has published more than 370 articles with a focus on neuromuscular disease and authored books on muscle disease, peripheral nerve disorders, and most recently, gene therapy for muscle disease. He was the principal investigator on the prednisone clinical trial in DMD in 1989 that has profoundly influenced clinical care. He was the PI on the exon skipping trial using eteplirsen, now FDA approved for clinical use in 2016. He was the PI on the first gene therapy trials for DMD and LGMD and was the PI on the SMA gene therapy trial that was FDA approved for systemic delivery for infants with SMA type 1 in 2017. The results of the first systemic gene therapy trial for DMD is now moving to Phase 3. Results from the Phase 1/2 micro-dystrophin trial will be published in June 2020.

Biography

Jerry R. Mendell, MD, is an attending neurologist at Nationwide Children’s Hospital, the Dwight E. Peters and Juanita R. Curran Endowed Chair in Pediatric Research at the Abigail Wexner Research Institute at Nationwide Children’s Hospital and professor of Pediatrics and Neurology at Nationwide Children’s and The Ohio State University. Dr. Mendell joined the Research Institute at Nationwide Children’s fulltime in 2004, recruited by Philip Johnson, the Head of the Center for Gene Therapy at that time. Shortly after, leadership of the Center changed and provided the opportunity to build and establish a program in Clinical Translation with emphasis on neuromuscular disease. At Nationwide Children’s, he holds academic positions as Professor of Pediatrics and Neurology and holds the Curran-Peters Chair of Pediatric Research. He is a clinician scientist with decades of experience. A career emphasis began with training in neuromuscular disease at the National Institutes of Health, directing laboratory projects that could be carried to the bedside. He was recruited to Columbus to direct the Neuromuscular Center at Ohio State. His life’s work has emphasized clinical translation. He has published more than 370 articles with a focus on neuromuscular disease and authored books on muscle disease, peripheral nerve disorders, and most recently, gene therapy for muscle disease. He was the principal investigator on the prednisone clinical trial in DMD in 1989 that has profoundly influenced clinical care. He was the PI on the exon skipping trial using eteplirsen, now FDA approved for clinical use in 2016. He was the PI on the first gene therapy trials for DMD and LGMD and was the PI on the SMA gene therapy trial that was FDA approved for systemic delivery for infants with SMA type 1 in 2017. The results of the first systemic gene therapy trial for DMD is now moving to Phase 3. Results from the Phase 1/2 micro-dystrophin trial will be published in June 2020.

Jerry R. Mendell, MD, is an attending neurologist at Nationwide Children’s Hospital, the Dwight E. Peters and Juanita R. Curran Endowed Chair in Pediatric Research at the Abigail Wexner Research Institute at Nationwide Children’s Hospital and professor of Pediatrics and Neurology at Nationwide Children’s and The Ohio State University. Dr. Mendell joined the Research Institute at Nationwide Children’s fulltime in 2004, recruited by Philip Johnson, the Head of the Center for Gene Therapy at that time. Shortly after, leadership of the Center changed and provided the opportunity to build and establish a program in Clinical Translation with emphasis on neuromuscular disease. At Nationwide Children’s, he holds academic positions as Professor of Pediatrics and Neurology and holds the Curran-Peters Chair of Pediatric Research. He is a clinician scientist with decades of experience. A career emphasis began with training in neuromuscular disease at the National Institutes of Health, directing laboratory projects that could be carried to the bedside. He was recruited to Columbus to direct the Neuromuscular Center at Ohio State. His life’s work has emphasized clinical translation. He has published more than 370 articles with a focus on neuromuscular disease and authored books on muscle disease, peripheral nerve disorders, and most recently, gene therapy for muscle disease. He was the principal investigator on the prednisone clinical trial in DMD in 1989 that has profoundly influenced clinical care. He was the PI on the exon skipping trial using eteplirsen, now FDA approved for clinical use in 2016. He was the PI on the first gene therapy trials for DMD and LGMD and was the PI on the SMA gene therapy trial that was FDA approved for systemic delivery for infants with SMA type 1 in 2017. The results of the first systemic gene therapy trial for DMD is now moving to Phase 3. Results from the Phase 1/2 micro-dystrophin trial will be published in June 2020.

Jerry R. Mendell, MD, is an attending neurologist at Nationwide Children’s Hospital, the Dwight E. Peters and Juanita R. Curran Endowed Chair in Pediatric Research at the Abigail Wexner Research Institute at Nationwide Children’s Hospital and professor of Pediatrics and Neurology at Nationwide Children’s and The Ohio State University.

Dr. Mendell joined the Research Institute at Nationwide Children’s fulltime in 2004, recruited by Philip Johnson, the Head of the Center for Gene Therapy at that time. Shortly after, leadership of the Center changed and provided the opportunity to build and establish a program in Clinical Translation with emphasis on neuromuscular disease. At Nationwide Children’s, he holds academic positions as Professor of Pediatrics and Neurology and holds the Curran-Peters Chair of Pediatric Research.

He is a clinician scientist with decades of experience. A career emphasis began with training in neuromuscular disease at the National Institutes of Health, directing laboratory projects that could be carried to the bedside. He was recruited to Columbus to direct the Neuromuscular Center at Ohio State. His life’s work has emphasized clinical translation. He has published more than 370 articles with a focus on neuromuscular disease and authored books on muscle disease, peripheral nerve disorders, and most recently, gene therapy for muscle disease. He was the principal investigator on the prednisone clinical trial in DMD in 1989 that has profoundly influenced clinical care. He was the PI on the exon skipping trial using eteplirsen, now FDA approved for clinical use in 2016. He was the PI on the first gene therapy trials for DMD and LGMD and was the PI on the SMA gene therapy trial that was FDA approved for systemic delivery for infants with SMA type 1 in 2017. The results of the first systemic gene therapy trial for DMD is now moving to Phase 3. Results from the Phase 1/2 micro-dystrophin trial will be published in June 2020.

Patient Care

Locations

Westerville Surgery Center

Main Campus of Nationwide Children’s Hospital

Patient Care

Locations

Westerville Surgery Center

Main Campus of Nationwide Children’s Hospital

Patient Care

Locations

Westerville Surgery Center

Main Campus of Nationwide Children’s Hospital

Locations

Westerville Surgery Center

Main Campus of Nationwide Children’s Hospital

Locations

Westerville Surgery Center

Main Campus of Nationwide Children’s Hospital

Locations

Westerville Surgery Center

Main Campus of Nationwide Children’s Hospital

• Westerville Surgery Center
• Main Campus of Nationwide Children’s Hospital

Academic and Clinical Areas

Neuromuscular Disorders

Physician Team

Neurology

Physician Team

Center for Gene Therapy

Principal Investigator

Neurology Residency

Faculty

Primary Department

Pediatrics

Primary Section

Neurology

Academic and Clinical Areas

Neuromuscular Disorders

Physician Team

Neurology

Physician Team

Center for Gene Therapy

Principal Investigator

Neurology Residency

Faculty

Primary Department

Pediatrics

Primary Section

Neurology

Academic and Clinical Areas

Neuromuscular Disorders

Physician Team

Neurology

Physician Team

Center for Gene Therapy

Principal Investigator

Neurology Residency

Faculty

Primary Department

Pediatrics

Primary Section

Neurology

Neuromuscular Disorders

Physician Team

Neurology

Physician Team

Center for Gene Therapy

Principal Investigator

Neurology Residency

Faculty

Primary Department

Pediatrics

Primary Section

Neurology

• Neuromuscular Disorders
• Physician Team
• Neurology
• Physician Team
• Center for Gene Therapy
• Principal Investigator
• Neurology Residency
• Faculty
• Primary Department
• Pediatrics
• Primary Section
• Neurology

Awards, Honors & Organizations

Juanita Curran and Dwight Peters Endowed Chair, 2010 Distinguished Scholar Award, The Ohio State University, Presented by the President, 2009 S. Mouchly Small Scientific Achievement Award, The Muscular Dystrophy Association for Significant Contributions to Neuromuscular Disease Research, 2004 Listed, Best Doctors in America

Awards, Honors & Organizations

Juanita Curran and Dwight Peters Endowed Chair, 2010 Distinguished Scholar Award, The Ohio State University, Presented by the President, 2009 S. Mouchly Small Scientific Achievement Award, The Muscular Dystrophy Association for Significant Contributions to Neuromuscular Disease Research, 2004 Listed, Best Doctors in America

Awards, Honors & Organizations

Juanita Curran and Dwight Peters Endowed Chair, 2010 Distinguished Scholar Award, The Ohio State University, Presented by the President, 2009 S. Mouchly Small Scientific Achievement Award, The Muscular Dystrophy Association for Significant Contributions to Neuromuscular Disease Research, 2004 Listed, Best Doctors in America

Juanita Curran and Dwight Peters Endowed Chair, 2010 Distinguished Scholar Award, The Ohio State University, Presented by the President, 2009 S. Mouchly Small Scientific Achievement Award, The Muscular Dystrophy Association for Significant Contributions to Neuromuscular Disease Research, 2004 Listed, Best Doctors in America

• Juanita Curran and Dwight Peters Endowed Chair, 2010
• Distinguished Scholar Award, The Ohio State University, Presented by the President, 2009
• S. Mouchly Small Scientific Achievement Award, The Muscular Dystrophy Association for Significant Contributions to Neuromuscular Disease Research, 2004
• Listed, Best Doctors in America

Research

Lab(s)

Center for Gene Therapy

The Mendell Lab is devoted to the development of potential treatments for neuromuscular diseases including Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophy (LGMD). DMD causes progressive muscle weakness that usually confines patients to a wheelchair in their early teens. Limb-girdle muscular dystrophies are a group of at least 13 diseases classified by their disease causing genes. It causes loss of muscle mass and strength in voluntary muscles, mainly those around the hips and shoulders. These diseases are caused by genetic defects that lead to missing or non-functional proteins. This makes gene replacement or gene therapy a good treatment option for these diseases. Our major research goal is successful gene therapy, by replacing genes for DMD and LGMD using adeno-associated virus (AAV) vectors to carry a copy of the corrected gene to the muscle cells. In some instances such as the dystrophin gene (the largest gene identified) “mini” and “micro” genes must be used. This can be done by direct injection into muscle (IM) or by way of the blood stream (vascular delivery). Muscular dystrophy can also be treated with follistatin by way of IM injection with adeno-associated virus (AAV) vectors to carry the follistatin gene in IBM and BMD. It inhibits myostatin, promoting muscle growth and preventing deterioration of existing muscle. Another therapeutic approach is exon skipping, treating primarily DMD, using synthetic molecules that induce skipping or read-through of the damaged portion of the dystrophin gene. This is a mutation-specific treatment with each molecule designed to skip a certain exon(s). This leads to restoration of the reading frame and production of an internally truncated partially functional dystrophin protein.In addition to developing potential treatments for neuromuscular diseases, the Mendell laboratory also concentrates on the diagnosis and characterization of neuromusclar diseases. The Mendell lab also serves as the diagnostic lab of muscle biopsies for the Anatomic Pathology Department of Nationwide Children’s Hospital. We are currently conducting a newborn screening study for DMD with The Research Institute at Nationwide Children’s Hospital and Cincinnati Children’s Hospital to identify possible cases in young males in the state of Ohio. The lab also has the resources of the United Dystrophinopathy Project (UDP) available for genetic screening and characterization of dystrophinopathies that cause DMD. The UDP directly sequences the coding and regulatory regions of the dystrophin gene, leading to faster and more specific characterization of mutations of many dystrophinopathy patients. The Mendell lab also has resources here at Nationwide Children’s Hospital to identify and sequence other genes that cause muscular dystrophies. Publications

                  Mitelman O, Abdel-Hamid HZ, Byrne BJ, Connolly AM, Heydemann P, Proud C, Shieh PB, Wagner KR, Dugar A, Santra S, Signorovitch J, Goemans N, investigators from the LNMRC Natural History study., McDonald CM, investigators from the CINRG Duchenne National History Study., Mercuri E, investigators from The DMD Italian Group., Mendell JR. A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirsen Treatment. J Neuromuscul Dis. 2022; 9: 39-52.

                


                  Day JW, Mendell JR, Mercuri E, Finkel RS, Strauss KA, Kleyn A, Tauscher-Wisniewski S, Tukov FF, Reyna SP, Chand DH. Correction to: Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy. Drug Saf. 2021 Dec 23;  

                


                  Sahenk Z, Ozes B, Murrey D, Myers M, Moss K, Yalvac ME, Ridgley A, Chen L, Mendell JR. Systemic delivery of AAVrh74.tMCK.hCAPN3 rescues the phenotype in a mouse model for LGMD2A/R1. Mol Ther Methods Clin Dev. 2021 Sep 10; 22: 401-414.

                


                  Al-Zaidy SA, Kolb SJ, Lowes L, Alfano LN, Shell R, Church KR, Nagendran S, Sproule DM, Feltner DE, Wells C, Ogrinc F, Menier M, L'Italien J, Arnold WD, Kissel JT, Kaspar BK, Mendell JR. AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort. J Neuromuscul Dis. 2019 Jul 30;  

                


                  Connolly AM, Zaidman CM, Golumbek PT, Cradock MM, Flanigan KM, Kuntz NL, Finkel RS, McDonald CM, Iannaccone ST, Anand P, Siener CA, Florence JM, Lowes LP, Alfano LN, Johnson LB, Nicorici A, Nelson LL, Mendell JR, MDA DMD Clinical Research Network.. Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy. Muscle Nerve. 2019 Jun; 59: 650-657.

                


                  Ke Q, Zhao ZY, Mendell JR, Baker M, Wiley V, Kwon JM, Alfano LN, Connolly AM, Jay C, Polari H, Ciafaloni E, Qi M, Griggs RC, Gatheridge MA. Progress in treatment and newborn screening for Duchenne muscular dystrophy and spinal muscular atrophy. World J Pediatr. 2019 Jun; 15: 219-225.

                


                  Al-Zaidy SA, Mendell JR. From Clinical Trials to Clinical Practice: Practical Considerations for Gene Replacement Therapy in SMA Type 1. Pediatr Neurol. 2019 Jun 13;  

                


                  Alfano LN, Charleston JS, Connolly AM, Cripe L, Donoghue C, Dracker R, Dworzak J, Eliopoulos H, Frank DE, Lewis S, Lucas K, Lynch J, Milici AJ, Flynt A, Naughton E, Rodino-Klapac LR, Sahenk Z, Schnell FJ, Young GD, Mendell JR, Lowes LP. Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy. Medicine (Baltimore). 2019 Jun; 98: e15858.

                


                  Lowes LP, Alfano LN, Arnold WD, Shell R, Prior TW, McColly M, Lehman KJ, Church K, Sproule DM, Nagendran S, Menier M, Feltner DE, Wells C, Kissel JT, Al-Zaidy S, Mendell J. Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy. Pediatr Neurol. 2019 May 13;  

                


                  Mendell JR, Chicoine LG, Al-Zaidy SA, Sahenk Z, Lehman K, Lowes L, Miller N, Alfano L, Galliers B, Lewis S, Murrey D, Peterson E, Griffin DA, Church K, Cheatham S, Cheatham J, Hogan MJ, Rodino-Klapac LR. Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion. Hum Gene Ther. 2019 Apr 19;  

                


                  Al-Zaidy S, Pickard AS, Kotha K, Alfano LN, Lowes L, Paul G, Church K, Lehman K, Sproule DM, Dabbous O, Maru B, Berry K, Arnold WD, Kissel JT, Mendell JR, Shell R. Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy. Pediatr Pulmonol. 2019 Feb; 54: 179-185.

                


                  Khan N, Eliopoulos H, Han L, Kinane TB, Lowes LP, Mendell JR, Gordish-Dressman H, Henricson EK, McDonald CM, Eteplirsen Investigators and the CINRG DNHS Investigators.. Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy. J Neuromuscul Dis. 2019; 6: 213-225.

                


                  Diaz-Manera J, Fernandez-Torron R, LLauger J, James MK, Mayhew A, Smith FE, Moore UR, Blamire AM, Carlier PG, Rufibach L, Mittal P, Eagle M, Jacobs M, Hodgson T, Wallace D, Ward L, Smith M, Stramare R, Rampado A, Sato N, Tamaru T, Harwick B, Rico Gala S, Turk S, Coppenrath EM, Foster G, Bendahan D, Le Fur Y, Fricke ST, Otero H, Foster SL, Peduto A, Sawyer AM, Hilsden H, Lochmuller H, Grieben U, Spuler S, Tesi Rocha C, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Harms M, Pestronk A, Krause S, Schreiber-Katz O, Walter MC, Paradas C, Hogrel JY, Stojkovic T, Takeda S, Mori-Yoshimura M, Bravver E, Sparks S, Bello L, Semplicini C, Pegoraro E, Mendell JR, Bushby K, Straub V, Jain COS Consortium.. Muscle MRI in patients with dysferlinopathy: pattern recognition and implications for clinical trials. J Neurol Neurosurg Psychiatry. 2018 Oct; 89: 1071-1081.

                


                  Mendell JR. Therapy for Spinal Muscular Atrophy. N Engl J Med. 2018 Feb 1; 378: 487.

                


                  Moore UR, Jacobs M, Fernandez-Torron R, Jang J, James MK, Mayhew A, Rufibach L, Mittal P, Eagle M, Cnaan A, Carlier PG, Blamire A, Hilsden H, Lochmüller H, Grieben U, Spuler S, Tesi Rocha C, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Harms M, Pestronk A, Krause S, Schreiber-Katz O, Walter MC, Paradas C, Hogrel JY, Stojkovic T, Takeda S, Mori-Yoshimura M, Bravver E, Sparks S, Diaz-Manera J, Bello L, Semplicini C, Pegoraro E, Mendell JR, Bushby K, Straub V. Teenage exercise is associated with earlier symptom onset in dysferlinopathy: a retrospective cohort study. J Neurol Neurosurg Psychiatry. 2018 Jan 29;  

                


                  Willcocks RJ, Triplett WT, Lott DJ, Forbes SC, Batra A, Sweeney HL, Mendell JR, Vandenborne K, Walter GA. Leg muscle MRI in identical twin boys with duchenne muscular dystrophy. Muscle Nerve. 2018 Jan 24;  

View More Publications

Research

Lab(s)

Center for Gene Therapy

The Mendell Lab is devoted to the development of potential treatments for neuromuscular diseases including Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophy (LGMD). DMD causes progressive muscle weakness that usually confines patients to a wheelchair in their early teens. Limb-girdle muscular dystrophies are a group of at least 13 diseases classified by their disease causing genes. It causes loss of muscle mass and strength in voluntary muscles, mainly those around the hips and shoulders. These diseases are caused by genetic defects that lead to missing or non-functional proteins. This makes gene replacement or gene therapy a good treatment option for these diseases. Our major research goal is successful gene therapy, by replacing genes for DMD and LGMD using adeno-associated virus (AAV) vectors to carry a copy of the corrected gene to the muscle cells. In some instances such as the dystrophin gene (the largest gene identified) “mini” and “micro” genes must be used. This can be done by direct injection into muscle (IM) or by way of the blood stream (vascular delivery). Muscular dystrophy can also be treated with follistatin by way of IM injection with adeno-associated virus (AAV) vectors to carry the follistatin gene in IBM and BMD. It inhibits myostatin, promoting muscle growth and preventing deterioration of existing muscle. Another therapeutic approach is exon skipping, treating primarily DMD, using synthetic molecules that induce skipping or read-through of the damaged portion of the dystrophin gene. This is a mutation-specific treatment with each molecule designed to skip a certain exon(s). This leads to restoration of the reading frame and production of an internally truncated partially functional dystrophin protein.In addition to developing potential treatments for neuromuscular diseases, the Mendell laboratory also concentrates on the diagnosis and characterization of neuromusclar diseases. The Mendell lab also serves as the diagnostic lab of muscle biopsies for the Anatomic Pathology Department of Nationwide Children’s Hospital. We are currently conducting a newborn screening study for DMD with The Research Institute at Nationwide Children’s Hospital and Cincinnati Children’s Hospital to identify possible cases in young males in the state of Ohio. The lab also has the resources of the United Dystrophinopathy Project (UDP) available for genetic screening and characterization of dystrophinopathies that cause DMD. The UDP directly sequences the coding and regulatory regions of the dystrophin gene, leading to faster and more specific characterization of mutations of many dystrophinopathy patients. The Mendell lab also has resources here at Nationwide Children’s Hospital to identify and sequence other genes that cause muscular dystrophies. Publications

                  Mitelman O, Abdel-Hamid HZ, Byrne BJ, Connolly AM, Heydemann P, Proud C, Shieh PB, Wagner KR, Dugar A, Santra S, Signorovitch J, Goemans N, investigators from the LNMRC Natural History study., McDonald CM, investigators from the CINRG Duchenne National History Study., Mercuri E, investigators from The DMD Italian Group., Mendell JR. A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirsen Treatment. J Neuromuscul Dis. 2022; 9: 39-52.

                


                  Day JW, Mendell JR, Mercuri E, Finkel RS, Strauss KA, Kleyn A, Tauscher-Wisniewski S, Tukov FF, Reyna SP, Chand DH. Correction to: Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy. Drug Saf. 2021 Dec 23;  

                


                  Sahenk Z, Ozes B, Murrey D, Myers M, Moss K, Yalvac ME, Ridgley A, Chen L, Mendell JR. Systemic delivery of AAVrh74.tMCK.hCAPN3 rescues the phenotype in a mouse model for LGMD2A/R1. Mol Ther Methods Clin Dev. 2021 Sep 10; 22: 401-414.

                


                  Al-Zaidy SA, Kolb SJ, Lowes L, Alfano LN, Shell R, Church KR, Nagendran S, Sproule DM, Feltner DE, Wells C, Ogrinc F, Menier M, L'Italien J, Arnold WD, Kissel JT, Kaspar BK, Mendell JR. AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort. J Neuromuscul Dis. 2019 Jul 30;  

                


                  Connolly AM, Zaidman CM, Golumbek PT, Cradock MM, Flanigan KM, Kuntz NL, Finkel RS, McDonald CM, Iannaccone ST, Anand P, Siener CA, Florence JM, Lowes LP, Alfano LN, Johnson LB, Nicorici A, Nelson LL, Mendell JR, MDA DMD Clinical Research Network.. Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy. Muscle Nerve. 2019 Jun; 59: 650-657.

                


                  Ke Q, Zhao ZY, Mendell JR, Baker M, Wiley V, Kwon JM, Alfano LN, Connolly AM, Jay C, Polari H, Ciafaloni E, Qi M, Griggs RC, Gatheridge MA. Progress in treatment and newborn screening for Duchenne muscular dystrophy and spinal muscular atrophy. World J Pediatr. 2019 Jun; 15: 219-225.

                


                  Al-Zaidy SA, Mendell JR. From Clinical Trials to Clinical Practice: Practical Considerations for Gene Replacement Therapy in SMA Type 1. Pediatr Neurol. 2019 Jun 13;  

                


                  Alfano LN, Charleston JS, Connolly AM, Cripe L, Donoghue C, Dracker R, Dworzak J, Eliopoulos H, Frank DE, Lewis S, Lucas K, Lynch J, Milici AJ, Flynt A, Naughton E, Rodino-Klapac LR, Sahenk Z, Schnell FJ, Young GD, Mendell JR, Lowes LP. Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy. Medicine (Baltimore). 2019 Jun; 98: e15858.

                


                  Lowes LP, Alfano LN, Arnold WD, Shell R, Prior TW, McColly M, Lehman KJ, Church K, Sproule DM, Nagendran S, Menier M, Feltner DE, Wells C, Kissel JT, Al-Zaidy S, Mendell J. Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy. Pediatr Neurol. 2019 May 13;  

                


                  Mendell JR, Chicoine LG, Al-Zaidy SA, Sahenk Z, Lehman K, Lowes L, Miller N, Alfano L, Galliers B, Lewis S, Murrey D, Peterson E, Griffin DA, Church K, Cheatham S, Cheatham J, Hogan MJ, Rodino-Klapac LR. Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion. Hum Gene Ther. 2019 Apr 19;  

                


                  Al-Zaidy S, Pickard AS, Kotha K, Alfano LN, Lowes L, Paul G, Church K, Lehman K, Sproule DM, Dabbous O, Maru B, Berry K, Arnold WD, Kissel JT, Mendell JR, Shell R. Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy. Pediatr Pulmonol. 2019 Feb; 54: 179-185.

                


                  Khan N, Eliopoulos H, Han L, Kinane TB, Lowes LP, Mendell JR, Gordish-Dressman H, Henricson EK, McDonald CM, Eteplirsen Investigators and the CINRG DNHS Investigators.. Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy. J Neuromuscul Dis. 2019; 6: 213-225.

                


                  Diaz-Manera J, Fernandez-Torron R, LLauger J, James MK, Mayhew A, Smith FE, Moore UR, Blamire AM, Carlier PG, Rufibach L, Mittal P, Eagle M, Jacobs M, Hodgson T, Wallace D, Ward L, Smith M, Stramare R, Rampado A, Sato N, Tamaru T, Harwick B, Rico Gala S, Turk S, Coppenrath EM, Foster G, Bendahan D, Le Fur Y, Fricke ST, Otero H, Foster SL, Peduto A, Sawyer AM, Hilsden H, Lochmuller H, Grieben U, Spuler S, Tesi Rocha C, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Harms M, Pestronk A, Krause S, Schreiber-Katz O, Walter MC, Paradas C, Hogrel JY, Stojkovic T, Takeda S, Mori-Yoshimura M, Bravver E, Sparks S, Bello L, Semplicini C, Pegoraro E, Mendell JR, Bushby K, Straub V, Jain COS Consortium.. Muscle MRI in patients with dysferlinopathy: pattern recognition and implications for clinical trials. J Neurol Neurosurg Psychiatry. 2018 Oct; 89: 1071-1081.

                


                  Mendell JR. Therapy for Spinal Muscular Atrophy. N Engl J Med. 2018 Feb 1; 378: 487.

                


                  Moore UR, Jacobs M, Fernandez-Torron R, Jang J, James MK, Mayhew A, Rufibach L, Mittal P, Eagle M, Cnaan A, Carlier PG, Blamire A, Hilsden H, Lochmüller H, Grieben U, Spuler S, Tesi Rocha C, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Harms M, Pestronk A, Krause S, Schreiber-Katz O, Walter MC, Paradas C, Hogrel JY, Stojkovic T, Takeda S, Mori-Yoshimura M, Bravver E, Sparks S, Diaz-Manera J, Bello L, Semplicini C, Pegoraro E, Mendell JR, Bushby K, Straub V. Teenage exercise is associated with earlier symptom onset in dysferlinopathy: a retrospective cohort study. J Neurol Neurosurg Psychiatry. 2018 Jan 29;  

                


                  Willcocks RJ, Triplett WT, Lott DJ, Forbes SC, Batra A, Sweeney HL, Mendell JR, Vandenborne K, Walter GA. Leg muscle MRI in identical twin boys with duchenne muscular dystrophy. Muscle Nerve. 2018 Jan 24;  

View More Publications

Research

Lab(s)

Center for Gene Therapy

The Mendell Lab is devoted to the development of potential treatments for neuromuscular diseases including Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophy (LGMD). DMD causes progressive muscle weakness that usually confines patients to a wheelchair in their early teens. Limb-girdle muscular dystrophies are a group of at least 13 diseases classified by their disease causing genes. It causes loss of muscle mass and strength in voluntary muscles, mainly those around the hips and shoulders. These diseases are caused by genetic defects that lead to missing or non-functional proteins. This makes gene replacement or gene therapy a good treatment option for these diseases. Our major research goal is successful gene therapy, by replacing genes for DMD and LGMD using adeno-associated virus (AAV) vectors to carry a copy of the corrected gene to the muscle cells. In some instances such as the dystrophin gene (the largest gene identified) “mini” and “micro” genes must be used. This can be done by direct injection into muscle (IM) or by way of the blood stream (vascular delivery). Muscular dystrophy can also be treated with follistatin by way of IM injection with adeno-associated virus (AAV) vectors to carry the follistatin gene in IBM and BMD. It inhibits myostatin, promoting muscle growth and preventing deterioration of existing muscle. Another therapeutic approach is exon skipping, treating primarily DMD, using synthetic molecules that induce skipping or read-through of the damaged portion of the dystrophin gene. This is a mutation-specific treatment with each molecule designed to skip a certain exon(s). This leads to restoration of the reading frame and production of an internally truncated partially functional dystrophin protein.In addition to developing potential treatments for neuromuscular diseases, the Mendell laboratory also concentrates on the diagnosis and characterization of neuromusclar diseases. The Mendell lab also serves as the diagnostic lab of muscle biopsies for the Anatomic Pathology Department of Nationwide Children’s Hospital. We are currently conducting a newborn screening study for DMD with The Research Institute at Nationwide Children’s Hospital and Cincinnati Children’s Hospital to identify possible cases in young males in the state of Ohio. The lab also has the resources of the United Dystrophinopathy Project (UDP) available for genetic screening and characterization of dystrophinopathies that cause DMD. The UDP directly sequences the coding and regulatory regions of the dystrophin gene, leading to faster and more specific characterization of mutations of many dystrophinopathy patients. The Mendell lab also has resources here at Nationwide Children’s Hospital to identify and sequence other genes that cause muscular dystrophies. Publications

                  Mitelman O, Abdel-Hamid HZ, Byrne BJ, Connolly AM, Heydemann P, Proud C, Shieh PB, Wagner KR, Dugar A, Santra S, Signorovitch J, Goemans N, investigators from the LNMRC Natural History study., McDonald CM, investigators from the CINRG Duchenne National History Study., Mercuri E, investigators from The DMD Italian Group., Mendell JR. A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirsen Treatment. J Neuromuscul Dis. 2022; 9: 39-52.

                


                  Day JW, Mendell JR, Mercuri E, Finkel RS, Strauss KA, Kleyn A, Tauscher-Wisniewski S, Tukov FF, Reyna SP, Chand DH. Correction to: Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy. Drug Saf. 2021 Dec 23;  

                


                  Sahenk Z, Ozes B, Murrey D, Myers M, Moss K, Yalvac ME, Ridgley A, Chen L, Mendell JR. Systemic delivery of AAVrh74.tMCK.hCAPN3 rescues the phenotype in a mouse model for LGMD2A/R1. Mol Ther Methods Clin Dev. 2021 Sep 10; 22: 401-414.

                


                  Al-Zaidy SA, Kolb SJ, Lowes L, Alfano LN, Shell R, Church KR, Nagendran S, Sproule DM, Feltner DE, Wells C, Ogrinc F, Menier M, L'Italien J, Arnold WD, Kissel JT, Kaspar BK, Mendell JR. AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort. J Neuromuscul Dis. 2019 Jul 30;  

                


                  Connolly AM, Zaidman CM, Golumbek PT, Cradock MM, Flanigan KM, Kuntz NL, Finkel RS, McDonald CM, Iannaccone ST, Anand P, Siener CA, Florence JM, Lowes LP, Alfano LN, Johnson LB, Nicorici A, Nelson LL, Mendell JR, MDA DMD Clinical Research Network.. Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy. Muscle Nerve. 2019 Jun; 59: 650-657.

                


                  Ke Q, Zhao ZY, Mendell JR, Baker M, Wiley V, Kwon JM, Alfano LN, Connolly AM, Jay C, Polari H, Ciafaloni E, Qi M, Griggs RC, Gatheridge MA. Progress in treatment and newborn screening for Duchenne muscular dystrophy and spinal muscular atrophy. World J Pediatr. 2019 Jun; 15: 219-225.

                


                  Al-Zaidy SA, Mendell JR. From Clinical Trials to Clinical Practice: Practical Considerations for Gene Replacement Therapy in SMA Type 1. Pediatr Neurol. 2019 Jun 13;  

                


                  Alfano LN, Charleston JS, Connolly AM, Cripe L, Donoghue C, Dracker R, Dworzak J, Eliopoulos H, Frank DE, Lewis S, Lucas K, Lynch J, Milici AJ, Flynt A, Naughton E, Rodino-Klapac LR, Sahenk Z, Schnell FJ, Young GD, Mendell JR, Lowes LP. Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy. Medicine (Baltimore). 2019 Jun; 98: e15858.

                


                  Lowes LP, Alfano LN, Arnold WD, Shell R, Prior TW, McColly M, Lehman KJ, Church K, Sproule DM, Nagendran S, Menier M, Feltner DE, Wells C, Kissel JT, Al-Zaidy S, Mendell J. Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy. Pediatr Neurol. 2019 May 13;  

                


                  Mendell JR, Chicoine LG, Al-Zaidy SA, Sahenk Z, Lehman K, Lowes L, Miller N, Alfano L, Galliers B, Lewis S, Murrey D, Peterson E, Griffin DA, Church K, Cheatham S, Cheatham J, Hogan MJ, Rodino-Klapac LR. Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion. Hum Gene Ther. 2019 Apr 19;  

                


                  Al-Zaidy S, Pickard AS, Kotha K, Alfano LN, Lowes L, Paul G, Church K, Lehman K, Sproule DM, Dabbous O, Maru B, Berry K, Arnold WD, Kissel JT, Mendell JR, Shell R. Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy. Pediatr Pulmonol. 2019 Feb; 54: 179-185.

                


                  Khan N, Eliopoulos H, Han L, Kinane TB, Lowes LP, Mendell JR, Gordish-Dressman H, Henricson EK, McDonald CM, Eteplirsen Investigators and the CINRG DNHS Investigators.. Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy. J Neuromuscul Dis. 2019; 6: 213-225.

                


                  Diaz-Manera J, Fernandez-Torron R, LLauger J, James MK, Mayhew A, Smith FE, Moore UR, Blamire AM, Carlier PG, Rufibach L, Mittal P, Eagle M, Jacobs M, Hodgson T, Wallace D, Ward L, Smith M, Stramare R, Rampado A, Sato N, Tamaru T, Harwick B, Rico Gala S, Turk S, Coppenrath EM, Foster G, Bendahan D, Le Fur Y, Fricke ST, Otero H, Foster SL, Peduto A, Sawyer AM, Hilsden H, Lochmuller H, Grieben U, Spuler S, Tesi Rocha C, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Harms M, Pestronk A, Krause S, Schreiber-Katz O, Walter MC, Paradas C, Hogrel JY, Stojkovic T, Takeda S, Mori-Yoshimura M, Bravver E, Sparks S, Bello L, Semplicini C, Pegoraro E, Mendell JR, Bushby K, Straub V, Jain COS Consortium.. Muscle MRI in patients with dysferlinopathy: pattern recognition and implications for clinical trials. J Neurol Neurosurg Psychiatry. 2018 Oct; 89: 1071-1081.

                


                  Mendell JR. Therapy for Spinal Muscular Atrophy. N Engl J Med. 2018 Feb 1; 378: 487.

                


                  Moore UR, Jacobs M, Fernandez-Torron R, Jang J, James MK, Mayhew A, Rufibach L, Mittal P, Eagle M, Cnaan A, Carlier PG, Blamire A, Hilsden H, Lochmüller H, Grieben U, Spuler S, Tesi Rocha C, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Harms M, Pestronk A, Krause S, Schreiber-Katz O, Walter MC, Paradas C, Hogrel JY, Stojkovic T, Takeda S, Mori-Yoshimura M, Bravver E, Sparks S, Diaz-Manera J, Bello L, Semplicini C, Pegoraro E, Mendell JR, Bushby K, Straub V. Teenage exercise is associated with earlier symptom onset in dysferlinopathy: a retrospective cohort study. J Neurol Neurosurg Psychiatry. 2018 Jan 29;  

                


                  Willcocks RJ, Triplett WT, Lott DJ, Forbes SC, Batra A, Sweeney HL, Mendell JR, Vandenborne K, Walter GA. Leg muscle MRI in identical twin boys with duchenne muscular dystrophy. Muscle Nerve. 2018 Jan 24;  

View More Publications

Lab(s)

Center for Gene Therapy

The Mendell Lab is devoted to the development of potential treatments for neuromuscular diseases including Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophy (LGMD). DMD causes progressive muscle weakness that usually confines patients to a wheelchair in their early teens. Limb-girdle muscular dystrophies are a group of at least 13 diseases classified by their disease causing genes. It causes loss of muscle mass and strength in voluntary muscles, mainly those around the hips and shoulders. These diseases are caused by genetic defects that lead to missing or non-functional proteins. This makes gene replacement or gene therapy a good treatment option for these diseases. Our major research goal is successful gene therapy, by replacing genes for DMD and LGMD using adeno-associated virus (AAV) vectors to carry a copy of the corrected gene to the muscle cells. In some instances such as the dystrophin gene (the largest gene identified) “mini” and “micro” genes must be used. This can be done by direct injection into muscle (IM) or by way of the blood stream (vascular delivery). Muscular dystrophy can also be treated with follistatin by way of IM injection with adeno-associated virus (AAV) vectors to carry the follistatin gene in IBM and BMD. It inhibits myostatin, promoting muscle growth and preventing deterioration of existing muscle. Another therapeutic approach is exon skipping, treating primarily DMD, using synthetic molecules that induce skipping or read-through of the damaged portion of the dystrophin gene. This is a mutation-specific treatment with each molecule designed to skip a certain exon(s). This leads to restoration of the reading frame and production of an internally truncated partially functional dystrophin protein.In addition to developing potential treatments for neuromuscular diseases, the Mendell laboratory also concentrates on the diagnosis and characterization of neuromusclar diseases. The Mendell lab also serves as the diagnostic lab of muscle biopsies for the Anatomic Pathology Department of Nationwide Children’s Hospital. We are currently conducting a newborn screening study for DMD with The Research Institute at Nationwide Children’s Hospital and Cincinnati Children’s Hospital to identify possible cases in young males in the state of Ohio. The lab also has the resources of the United Dystrophinopathy Project (UDP) available for genetic screening and characterization of dystrophinopathies that cause DMD. The UDP directly sequences the coding and regulatory regions of the dystrophin gene, leading to faster and more specific characterization of mutations of many dystrophinopathy patients. The Mendell lab also has resources here at Nationwide Children’s Hospital to identify and sequence other genes that cause muscular dystrophies. Publications

                  Mitelman O, Abdel-Hamid HZ, Byrne BJ, Connolly AM, Heydemann P, Proud C, Shieh PB, Wagner KR, Dugar A, Santra S, Signorovitch J, Goemans N, investigators from the LNMRC Natural History study., McDonald CM, investigators from the CINRG Duchenne National History Study., Mercuri E, investigators from The DMD Italian Group., Mendell JR. A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirsen Treatment. J Neuromuscul Dis. 2022; 9: 39-52.

                


                  Day JW, Mendell JR, Mercuri E, Finkel RS, Strauss KA, Kleyn A, Tauscher-Wisniewski S, Tukov FF, Reyna SP, Chand DH. Correction to: Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy. Drug Saf. 2021 Dec 23;  

                


                  Sahenk Z, Ozes B, Murrey D, Myers M, Moss K, Yalvac ME, Ridgley A, Chen L, Mendell JR. Systemic delivery of AAVrh74.tMCK.hCAPN3 rescues the phenotype in a mouse model for LGMD2A/R1. Mol Ther Methods Clin Dev. 2021 Sep 10; 22: 401-414.

                


                  Al-Zaidy SA, Kolb SJ, Lowes L, Alfano LN, Shell R, Church KR, Nagendran S, Sproule DM, Feltner DE, Wells C, Ogrinc F, Menier M, L'Italien J, Arnold WD, Kissel JT, Kaspar BK, Mendell JR. AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort. J Neuromuscul Dis. 2019 Jul 30;  

                


                  Connolly AM, Zaidman CM, Golumbek PT, Cradock MM, Flanigan KM, Kuntz NL, Finkel RS, McDonald CM, Iannaccone ST, Anand P, Siener CA, Florence JM, Lowes LP, Alfano LN, Johnson LB, Nicorici A, Nelson LL, Mendell JR, MDA DMD Clinical Research Network.. Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy. Muscle Nerve. 2019 Jun; 59: 650-657.

                


                  Ke Q, Zhao ZY, Mendell JR, Baker M, Wiley V, Kwon JM, Alfano LN, Connolly AM, Jay C, Polari H, Ciafaloni E, Qi M, Griggs RC, Gatheridge MA. Progress in treatment and newborn screening for Duchenne muscular dystrophy and spinal muscular atrophy. World J Pediatr. 2019 Jun; 15: 219-225.

                


                  Al-Zaidy SA, Mendell JR. From Clinical Trials to Clinical Practice: Practical Considerations for Gene Replacement Therapy in SMA Type 1. Pediatr Neurol. 2019 Jun 13;  

                


                  Alfano LN, Charleston JS, Connolly AM, Cripe L, Donoghue C, Dracker R, Dworzak J, Eliopoulos H, Frank DE, Lewis S, Lucas K, Lynch J, Milici AJ, Flynt A, Naughton E, Rodino-Klapac LR, Sahenk Z, Schnell FJ, Young GD, Mendell JR, Lowes LP. Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy. Medicine (Baltimore). 2019 Jun; 98: e15858.

                


                  Lowes LP, Alfano LN, Arnold WD, Shell R, Prior TW, McColly M, Lehman KJ, Church K, Sproule DM, Nagendran S, Menier M, Feltner DE, Wells C, Kissel JT, Al-Zaidy S, Mendell J. Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy. Pediatr Neurol. 2019 May 13;  

                


                  Mendell JR, Chicoine LG, Al-Zaidy SA, Sahenk Z, Lehman K, Lowes L, Miller N, Alfano L, Galliers B, Lewis S, Murrey D, Peterson E, Griffin DA, Church K, Cheatham S, Cheatham J, Hogan MJ, Rodino-Klapac LR. Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion. Hum Gene Ther. 2019 Apr 19;  

                


                  Al-Zaidy S, Pickard AS, Kotha K, Alfano LN, Lowes L, Paul G, Church K, Lehman K, Sproule DM, Dabbous O, Maru B, Berry K, Arnold WD, Kissel JT, Mendell JR, Shell R. Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy. Pediatr Pulmonol. 2019 Feb; 54: 179-185.

                


                  Khan N, Eliopoulos H, Han L, Kinane TB, Lowes LP, Mendell JR, Gordish-Dressman H, Henricson EK, McDonald CM, Eteplirsen Investigators and the CINRG DNHS Investigators.. Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy. J Neuromuscul Dis. 2019; 6: 213-225.

                


                  Diaz-Manera J, Fernandez-Torron R, LLauger J, James MK, Mayhew A, Smith FE, Moore UR, Blamire AM, Carlier PG, Rufibach L, Mittal P, Eagle M, Jacobs M, Hodgson T, Wallace D, Ward L, Smith M, Stramare R, Rampado A, Sato N, Tamaru T, Harwick B, Rico Gala S, Turk S, Coppenrath EM, Foster G, Bendahan D, Le Fur Y, Fricke ST, Otero H, Foster SL, Peduto A, Sawyer AM, Hilsden H, Lochmuller H, Grieben U, Spuler S, Tesi Rocha C, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Harms M, Pestronk A, Krause S, Schreiber-Katz O, Walter MC, Paradas C, Hogrel JY, Stojkovic T, Takeda S, Mori-Yoshimura M, Bravver E, Sparks S, Bello L, Semplicini C, Pegoraro E, Mendell JR, Bushby K, Straub V, Jain COS Consortium.. Muscle MRI in patients with dysferlinopathy: pattern recognition and implications for clinical trials. J Neurol Neurosurg Psychiatry. 2018 Oct; 89: 1071-1081.

                


                  Mendell JR. Therapy for Spinal Muscular Atrophy. N Engl J Med. 2018 Feb 1; 378: 487.

                


                  Moore UR, Jacobs M, Fernandez-Torron R, Jang J, James MK, Mayhew A, Rufibach L, Mittal P, Eagle M, Cnaan A, Carlier PG, Blamire A, Hilsden H, Lochmüller H, Grieben U, Spuler S, Tesi Rocha C, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Harms M, Pestronk A, Krause S, Schreiber-Katz O, Walter MC, Paradas C, Hogrel JY, Stojkovic T, Takeda S, Mori-Yoshimura M, Bravver E, Sparks S, Diaz-Manera J, Bello L, Semplicini C, Pegoraro E, Mendell JR, Bushby K, Straub V. Teenage exercise is associated with earlier symptom onset in dysferlinopathy: a retrospective cohort study. J Neurol Neurosurg Psychiatry. 2018 Jan 29;  

                


                  Willcocks RJ, Triplett WT, Lott DJ, Forbes SC, Batra A, Sweeney HL, Mendell JR, Vandenborne K, Walter GA. Leg muscle MRI in identical twin boys with duchenne muscular dystrophy. Muscle Nerve. 2018 Jan 24;  

View More Publications

Lab(s)

Center for Gene Therapy

The Mendell Lab is devoted to the development of potential treatments for neuromuscular diseases including Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophy (LGMD). DMD causes progressive muscle weakness that usually confines patients to a wheelchair in their early teens. Limb-girdle muscular dystrophies are a group of at least 13 diseases classified by their disease causing genes. It causes loss of muscle mass and strength in voluntary muscles, mainly those around the hips and shoulders. These diseases are caused by genetic defects that lead to missing or non-functional proteins. This makes gene replacement or gene therapy a good treatment option for these diseases. Our major research goal is successful gene therapy, by replacing genes for DMD and LGMD using adeno-associated virus (AAV) vectors to carry a copy of the corrected gene to the muscle cells. In some instances such as the dystrophin gene (the largest gene identified) “mini” and “micro” genes must be used. This can be done by direct injection into muscle (IM) or by way of the blood stream (vascular delivery). Muscular dystrophy can also be treated with follistatin by way of IM injection with adeno-associated virus (AAV) vectors to carry the follistatin gene in IBM and BMD. It inhibits myostatin, promoting muscle growth and preventing deterioration of existing muscle. Another therapeutic approach is exon skipping, treating primarily DMD, using synthetic molecules that induce skipping or read-through of the damaged portion of the dystrophin gene. This is a mutation-specific treatment with each molecule designed to skip a certain exon(s). This leads to restoration of the reading frame and production of an internally truncated partially functional dystrophin protein.In addition to developing potential treatments for neuromuscular diseases, the Mendell laboratory also concentrates on the diagnosis and characterization of neuromusclar diseases. The Mendell lab also serves as the diagnostic lab of muscle biopsies for the Anatomic Pathology Department of Nationwide Children’s Hospital. We are currently conducting a newborn screening study for DMD with The Research Institute at Nationwide Children’s Hospital and Cincinnati Children’s Hospital to identify possible cases in young males in the state of Ohio. The lab also has the resources of the United Dystrophinopathy Project (UDP) available for genetic screening and characterization of dystrophinopathies that cause DMD. The UDP directly sequences the coding and regulatory regions of the dystrophin gene, leading to faster and more specific characterization of mutations of many dystrophinopathy patients. The Mendell lab also has resources here at Nationwide Children’s Hospital to identify and sequence other genes that cause muscular dystrophies.

Lab(s)

Center for Gene Therapy

• Center for Gene Therapy

                  Mitelman O, Abdel-Hamid HZ, Byrne BJ, Connolly AM, Heydemann P, Proud C, Shieh PB, Wagner KR, Dugar A, Santra S, Signorovitch J, Goemans N, investigators from the LNMRC Natural History study., McDonald CM, investigators from the CINRG Duchenne National History Study., Mercuri E, investigators from The DMD Italian Group., Mendell JR. A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirsen Treatment. J Neuromuscul Dis. 2022; 9: 39-52.
  
  
  
                  Day JW, Mendell JR, Mercuri E, Finkel RS, Strauss KA, Kleyn A, Tauscher-Wisniewski S, Tukov FF, Reyna SP, Chand DH. Correction to: Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy. Drug Saf. 2021 Dec 23;  
  
  
  
                  Sahenk Z, Ozes B, Murrey D, Myers M, Moss K, Yalvac ME, Ridgley A, Chen L, Mendell JR. Systemic delivery of AAVrh74.tMCK.hCAPN3 rescues the phenotype in a mouse model for LGMD2A/R1. Mol Ther Methods Clin Dev. 2021 Sep 10; 22: 401-414.
  
  
  
                  Al-Zaidy SA, Kolb SJ, Lowes L, Alfano LN, Shell R, Church KR, Nagendran S, Sproule DM, Feltner DE, Wells C, Ogrinc F, Menier M, L'Italien J, Arnold WD, Kissel JT, Kaspar BK, Mendell JR. AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort. J Neuromuscul Dis. 2019 Jul 30;  
  
  
  
                  Connolly AM, Zaidman CM, Golumbek PT, Cradock MM, Flanigan KM, Kuntz NL, Finkel RS, McDonald CM, Iannaccone ST, Anand P, Siener CA, Florence JM, Lowes LP, Alfano LN, Johnson LB, Nicorici A, Nelson LL, Mendell JR, MDA DMD Clinical Research Network.. Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy. Muscle Nerve. 2019 Jun; 59: 650-657.
  
  
  
                  Ke Q, Zhao ZY, Mendell JR, Baker M, Wiley V, Kwon JM, Alfano LN, Connolly AM, Jay C, Polari H, Ciafaloni E, Qi M, Griggs RC, Gatheridge MA. Progress in treatment and newborn screening for Duchenne muscular dystrophy and spinal muscular atrophy. World J Pediatr. 2019 Jun; 15: 219-225.
  
  
  
                  Al-Zaidy SA, Mendell JR. From Clinical Trials to Clinical Practice: Practical Considerations for Gene Replacement Therapy in SMA Type 1. Pediatr Neurol. 2019 Jun 13;  
  
  
  
                  Alfano LN, Charleston JS, Connolly AM, Cripe L, Donoghue C, Dracker R, Dworzak J, Eliopoulos H, Frank DE, Lewis S, Lucas K, Lynch J, Milici AJ, Flynt A, Naughton E, Rodino-Klapac LR, Sahenk Z, Schnell FJ, Young GD, Mendell JR, Lowes LP. Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy. Medicine (Baltimore). 2019 Jun; 98: e15858.
  
  
  
                  Lowes LP, Alfano LN, Arnold WD, Shell R, Prior TW, McColly M, Lehman KJ, Church K, Sproule DM, Nagendran S, Menier M, Feltner DE, Wells C, Kissel JT, Al-Zaidy S, Mendell J. Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy. Pediatr Neurol. 2019 May 13;  
  
  
  
                  Mendell JR, Chicoine LG, Al-Zaidy SA, Sahenk Z, Lehman K, Lowes L, Miller N, Alfano L, Galliers B, Lewis S, Murrey D, Peterson E, Griffin DA, Church K, Cheatham S, Cheatham J, Hogan MJ, Rodino-Klapac LR. Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion. Hum Gene Ther. 2019 Apr 19;  
  
  
  
                  Al-Zaidy S, Pickard AS, Kotha K, Alfano LN, Lowes L, Paul G, Church K, Lehman K, Sproule DM, Dabbous O, Maru B, Berry K, Arnold WD, Kissel JT, Mendell JR, Shell R. Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy. Pediatr Pulmonol. 2019 Feb; 54: 179-185.
  
  
  
                  Khan N, Eliopoulos H, Han L, Kinane TB, Lowes LP, Mendell JR, Gordish-Dressman H, Henricson EK, McDonald CM, Eteplirsen Investigators and the CINRG DNHS Investigators.. Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy. J Neuromuscul Dis. 2019; 6: 213-225.
  
  
  
                  Diaz-Manera J, Fernandez-Torron R, LLauger J, James MK, Mayhew A, Smith FE, Moore UR, Blamire AM, Carlier PG, Rufibach L, Mittal P, Eagle M, Jacobs M, Hodgson T, Wallace D, Ward L, Smith M, Stramare R, Rampado A, Sato N, Tamaru T, Harwick B, Rico Gala S, Turk S, Coppenrath EM, Foster G, Bendahan D, Le Fur Y, Fricke ST, Otero H, Foster SL, Peduto A, Sawyer AM, Hilsden H, Lochmuller H, Grieben U, Spuler S, Tesi Rocha C, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Harms M, Pestronk A, Krause S, Schreiber-Katz O, Walter MC, Paradas C, Hogrel JY, Stojkovic T, Takeda S, Mori-Yoshimura M, Bravver E, Sparks S, Bello L, Semplicini C, Pegoraro E, Mendell JR, Bushby K, Straub V, Jain COS Consortium.. Muscle MRI in patients with dysferlinopathy: pattern recognition and implications for clinical trials. J Neurol Neurosurg Psychiatry. 2018 Oct; 89: 1071-1081.
  
  
  
                  Mendell JR. Therapy for Spinal Muscular Atrophy. N Engl J Med. 2018 Feb 1; 378: 487.
  
  
  
                  Moore UR, Jacobs M, Fernandez-Torron R, Jang J, James MK, Mayhew A, Rufibach L, Mittal P, Eagle M, Cnaan A, Carlier PG, Blamire A, Hilsden H, Lochmüller H, Grieben U, Spuler S, Tesi Rocha C, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Harms M, Pestronk A, Krause S, Schreiber-Katz O, Walter MC, Paradas C, Hogrel JY, Stojkovic T, Takeda S, Mori-Yoshimura M, Bravver E, Sparks S, Diaz-Manera J, Bello L, Semplicini C, Pegoraro E, Mendell JR, Bushby K, Straub V. Teenage exercise is associated with earlier symptom onset in dysferlinopathy: a retrospective cohort study. J Neurol Neurosurg Psychiatry. 2018 Jan 29;  
  
  
  
                  Willcocks RJ, Triplett WT, Lott DJ, Forbes SC, Batra A, Sweeney HL, Mendell JR, Vandenborne K, Walter GA. Leg muscle MRI in identical twin boys with duchenne muscular dystrophy. Muscle Nerve. 2018 Jan 24;  
  
  

View More Publications

• Mitelman O, Abdel-Hamid HZ, Byrne BJ, Connolly AM, Heydemann P, Proud C, Shieh PB, Wagner KR, Dugar A, Santra S, Signorovitch J, Goemans N, investigators from the LNMRC Natural History study., McDonald CM, investigators from the CINRG Duchenne National History Study., Mercuri E, investigators from The DMD Italian Group., Mendell JR. A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirsen Treatment. J Neuromuscul Dis. 2022; 9: 39-52.
• Day JW, Mendell JR, Mercuri E, Finkel RS, Strauss KA, Kleyn A, Tauscher-Wisniewski S, Tukov FF, Reyna SP, Chand DH. Correction to: Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy. Drug Saf. 2021 Dec 23;
• Sahenk Z, Ozes B, Murrey D, Myers M, Moss K, Yalvac ME, Ridgley A, Chen L, Mendell JR. Systemic delivery of AAVrh74.tMCK.hCAPN3 rescues the phenotype in a mouse model for LGMD2A/R1. Mol Ther Methods Clin Dev. 2021 Sep 10; 22: 401-414.
• Al-Zaidy SA, Kolb SJ, Lowes L, Alfano LN, Shell R, Church KR, Nagendran S, Sproule DM, Feltner DE, Wells C, Ogrinc F, Menier M, L’Italien J, Arnold WD, Kissel JT, Kaspar BK, Mendell JR. AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort. J Neuromuscul Dis. 2019 Jul 30;
• Connolly AM, Zaidman CM, Golumbek PT, Cradock MM, Flanigan KM, Kuntz NL, Finkel RS, McDonald CM, Iannaccone ST, Anand P, Siener CA, Florence JM, Lowes LP, Alfano LN, Johnson LB, Nicorici A, Nelson LL, Mendell JR, MDA DMD Clinical Research Network.. Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy. Muscle Nerve. 2019 Jun; 59: 650-657.
• Ke Q, Zhao ZY, Mendell JR, Baker M, Wiley V, Kwon JM, Alfano LN, Connolly AM, Jay C, Polari H, Ciafaloni E, Qi M, Griggs RC, Gatheridge MA. Progress in treatment and newborn screening for Duchenne muscular dystrophy and spinal muscular atrophy. World J Pediatr. 2019 Jun; 15: 219-225.
• Al-Zaidy SA, Mendell JR. From Clinical Trials to Clinical Practice: Practical Considerations for Gene Replacement Therapy in SMA Type 1. Pediatr Neurol. 2019 Jun 13;
• Alfano LN, Charleston JS, Connolly AM, Cripe L, Donoghue C, Dracker R, Dworzak J, Eliopoulos H, Frank DE, Lewis S, Lucas K, Lynch J, Milici AJ, Flynt A, Naughton E, Rodino-Klapac LR, Sahenk Z, Schnell FJ, Young GD, Mendell JR, Lowes LP. Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy. Medicine (Baltimore). 2019 Jun; 98: e15858.
• Lowes LP, Alfano LN, Arnold WD, Shell R, Prior TW, McColly M, Lehman KJ, Church K, Sproule DM, Nagendran S, Menier M, Feltner DE, Wells C, Kissel JT, Al-Zaidy S, Mendell J. Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy. Pediatr Neurol. 2019 May 13;
• Mendell JR, Chicoine LG, Al-Zaidy SA, Sahenk Z, Lehman K, Lowes L, Miller N, Alfano L, Galliers B, Lewis S, Murrey D, Peterson E, Griffin DA, Church K, Cheatham S, Cheatham J, Hogan MJ, Rodino-Klapac LR. Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion. Hum Gene Ther. 2019 Apr 19;
• Al-Zaidy S, Pickard AS, Kotha K, Alfano LN, Lowes L, Paul G, Church K, Lehman K, Sproule DM, Dabbous O, Maru B, Berry K, Arnold WD, Kissel JT, Mendell JR, Shell R. Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy. Pediatr Pulmonol. 2019 Feb; 54: 179-185.
• Khan N, Eliopoulos H, Han L, Kinane TB, Lowes LP, Mendell JR, Gordish-Dressman H, Henricson EK, McDonald CM, Eteplirsen Investigators and the CINRG DNHS Investigators.. Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy. J Neuromuscul Dis. 2019; 6: 213-225.
• Diaz-Manera J, Fernandez-Torron R, LLauger J, James MK, Mayhew A, Smith FE, Moore UR, Blamire AM, Carlier PG, Rufibach L, Mittal P, Eagle M, Jacobs M, Hodgson T, Wallace D, Ward L, Smith M, Stramare R, Rampado A, Sato N, Tamaru T, Harwick B, Rico Gala S, Turk S, Coppenrath EM, Foster G, Bendahan D, Le Fur Y, Fricke ST, Otero H, Foster SL, Peduto A, Sawyer AM, Hilsden H, Lochmuller H, Grieben U, Spuler S, Tesi Rocha C, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Harms M, Pestronk A, Krause S, Schreiber-Katz O, Walter MC, Paradas C, Hogrel JY, Stojkovic T, Takeda S, Mori-Yoshimura M, Bravver E, Sparks S, Bello L, Semplicini C, Pegoraro E, Mendell JR, Bushby K, Straub V, Jain COS Consortium.. Muscle MRI in patients with dysferlinopathy: pattern recognition and implications for clinical trials. J Neurol Neurosurg Psychiatry. 2018 Oct; 89: 1071-1081.
• Mendell JR. Therapy for Spinal Muscular Atrophy. N Engl J Med. 2018 Feb 1; 378: 487.
• Moore UR, Jacobs M, Fernandez-Torron R, Jang J, James MK, Mayhew A, Rufibach L, Mittal P, Eagle M, Cnaan A, Carlier PG, Blamire A, Hilsden H, Lochmüller H, Grieben U, Spuler S, Tesi Rocha C, Day JW, Jones KJ, Bharucha-Goebel DX, Salort-Campana E, Harms M, Pestronk A, Krause S, Schreiber-Katz O, Walter MC, Paradas C, Hogrel JY, Stojkovic T, Takeda S, Mori-Yoshimura M, Bravver E, Sparks S, Diaz-Manera J, Bello L, Semplicini C, Pegoraro E, Mendell JR, Bushby K, Straub V. Teenage exercise is associated with earlier symptom onset in dysferlinopathy: a retrospective cohort study. J Neurol Neurosurg Psychiatry. 2018 Jan 29;
• Willcocks RJ, Triplett WT, Lott DJ, Forbes SC, Batra A, Sweeney HL, Mendell JR, Vandenborne K, Walter GA. Leg muscle MRI in identical twin boys with duchenne muscular dystrophy. Muscle Nerve. 2018 Jan 24;

Education

Date of Appointment at Nationwide Children’s Hospital: 08/27/2004

Board Certifications

Neurology

Fellowship

National Institutes of Health

Date Completed: 07/31/1971

Residency

National Institutes of Health

Date Completed: 06/30/1970

Residency

Columbia Presbyterian Medical Center - New York

Date Completed: 06/30/1969

Medical School

University of Texas Southwestern Medical Center

Date Completed: 06/30/1966

Education

Date of Appointment at Nationwide Children’s Hospital: 08/27/2004

Board Certifications

Neurology

Fellowship

National Institutes of Health

Date Completed: 07/31/1971

Residency

National Institutes of Health

Date Completed: 06/30/1970

Residency

Columbia Presbyterian Medical Center - New York

Date Completed: 06/30/1969

Medical School

University of Texas Southwestern Medical Center

Date Completed: 06/30/1966

Education

Date of Appointment at Nationwide Children’s Hospital: 08/27/2004

Board Certifications

Neurology

Fellowship

National Institutes of Health

Date Completed: 07/31/1971

Residency

National Institutes of Health

Date Completed: 06/30/1970

Residency

Columbia Presbyterian Medical Center - New York

Date Completed: 06/30/1969

Medical School

University of Texas Southwestern Medical Center

Date Completed: 06/30/1966

Date of Appointment at Nationwide Children’s Hospital: 08/27/2004

Board Certifications

Neurology

Fellowship

National Institutes of Health

Date Completed: 07/31/1971

Residency

National Institutes of Health

Date Completed: 06/30/1970

Residency

Columbia Presbyterian Medical Center - New York

Date Completed: 06/30/1969

Medical School

University of Texas Southwestern Medical Center

Date Completed: 06/30/1966

Date of Appointment at Nationwide Children’s Hospital: 08/27/2004

Board Certifications

Neurology

Fellowship

National Institutes of Health

Date Completed: 07/31/1971

Residency

National Institutes of Health

Date Completed: 06/30/1970

Residency

Columbia Presbyterian Medical Center - New York

Date Completed: 06/30/1969

Medical School

University of Texas Southwestern Medical Center

Date Completed: 06/30/1966

• Neurology

Professional Experience

1992 - 2004 Dept of Neurology, The Ohio State University, Chair

Professional Experience

1992 - 2004 Dept of Neurology, The Ohio State University, Chair

Professional Experience

1992 - 2004 Dept of Neurology, The Ohio State University, Chair

1992 - 2004 Dept of Neurology, The Ohio State University, Chair

1992 - 2004 Dept of Neurology, The Ohio State University, Chair

Contact Information

Pediatrics

Call us at: (614) 722-4877

Fax us at: (614) 355-7686

                    700 Children's DrColumbus, OH 43205 (map)

Contact Information

Pediatrics

Call us at: (614) 722-4877

Fax us at: (614) 355-7686

                    700 Children's DrColumbus, OH 43205 (map)

Contact Information

Pediatrics

Call us at: (614) 722-4877

Fax us at: (614) 355-7686

                    700 Children's DrColumbus, OH 43205 (map)

Pediatrics

Call us at: (614) 722-4877

Fax us at: (614) 355-7686

                    700 Children's DrColumbus, OH 43205 (map)

Call us at: (614) 722-4877

Fax us at: (614) 355-7686

                    700 Children's DrColumbus, OH 43205 (map)

Call us at: (614) 722-4877

Fax us at: (614) 355-7686

                    700 Children's DrColumbus, OH 43205 (map)

• Call us at:
• (614) 722-4877
• Fax us at:
• (614) 355-7686
• 700 Children’s DrColumbus, OH 43205 (map)