Zarife Sahenk

Contact Information

Call us at: (614) 722-2203

Fax us at: (614) 355-5247

PediatricsNeurology700 Children’s DrColumbus, OH 43205 (map)

Learn more about Zarife Sahenk

Biography

Zarife Sahenk, MD, PhD, is an attending neurologist at Nationwide Children’s, Director of Clinical and Experimental Neuromuscular Pathology at The Research Institute at Nationwide Children’s and Professor of Pediatrics, Pathology and Neurology at The Ohio State University College of Medicine. Her laboratory plays an integral part in the preclinical studies and the gene therapy clinical trials that take place in the Center for Gene Therapy at Nationwide Children’s Hospital. She has provided valuable analysis of muscle derived from the first clinical gene trials for Duchenne muscular dystrophy, Limb Girdle muscular dystrophy, and follistatin gene therapy for Becker muscular dystrophy, and inclusion body myositis. A primary goal of her research program is to gain a detailed understanding of the anatomical and molecular basis of nerve degeneration and impaired regeneration in hereditary peripheral neuropathies. Particular emphasis is placed on the role of trophic factors secreted by Schwann cells in regeneration-associated myelination. The role of neurotrophin-3 (NT-3) in nerve regeneration in animal models of Charcot-Marie-Tooth (CMT) is currently being investigated and a gene therapy paradigm is being developed using AAV1.NT-3. In addition, a novel translational therapeutic approach that combines vasoactive intestinal peptide (VIP), a lentivirus vector and bone marrow derived dendritic cells for the treatment of chronic inflammatory demyelinating polyradiculoneuropathy is currently being developed in her laboratory. Dr. Sahenk is named among the Best Doctors in America.

Patient Care

Locations

Westerville Surgery Center

Main Campus of Nationwide Children’s Hospital

Academic and Clinical Areas

Neurology

Physician Team

Neuromuscular Disorders

Physician Team

Center for Gene Therapy

Principal Investigator

Neurology Residency

Faculty

Primary Department

Pediatrics

Primary Section

Neurology

Awards, Honors & Organizations

Listed, Best Doctors in America

Research

Lab(s)

Center for Gene Therapy

A primary goal of Dr. Sahenk’s research program is to gain a detailed understanding of the anatomical and molecular basis of hereditary peripheral neuropathies. Particular emphasis is placed on the role of trophic factors secreted by Schwann cells in regeneration-associated myelination. The role of neurotrophin-3 in nerve regeneration in patients and animal models of Charcot-Marie-Tooth (CMT) is currently being investigated. These studies are relevant to understanding the mechanism of axonal loss and impaired regeneration in CMT disorders and developing rational therapies to promote nerve repair. Publications

                  Pozsgai E, Griffin D, Potter R, Sahenk Z, Lehman K, Rodino-Klapac LR, Mendell JR. Unmet needs and evolving treatment for limb girdle muscular dystrophies. Neurodegener Dis Manag. 2021 Oct; 11: 411-429.

                


                  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.

                


                  Jiang B, Sahenk Z, Satoskar A, Freimer M, Ayoub I. Vasculitic neuropathy associated with IgG4-related kidney disease: A case report and literature review. Clin Nephrol. 2021 Sep; 96: 175-179.

                


                  Ozes B, Myers M, Moss K, Mckinney J, Ridgley A, Chen L, Bai S, Abrams CK, Freidin MM, Mendell JR, Sahenk Z. AAV1.NT-3 gene therapy for X-linked Charcot-Marie-Tooth neuropathy type 1. Gene Ther. 2021 Feb 4;  

                


                  Ozes B, Moss K, Myers M, Ridgley A, Chen L, Murrey D, Sahenk Z. AAV1.NT-3 gene therapy in a CMT2D model: phenotypic improvements in GarsP278KY/+ mice. Brain Commun. 2021; 3: fcab252.

                


                  Mendell JR, Sahenk Z, Lehman K, Nease C, Lowes LP, Miller NF, Iammarino MA, Alfano LN, Nicholl A, Al-Zaidy S, Lewis S, Church K, Shell R, Cripe LH, Potter RA, Griffin DA, Pozsgai E, Dugar A, Hogan M, Rodino-Klapac LR. Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial. JAMA Neurol. 2020 Jun 15;  

                


                  Sahenk Z, Ak BO. Gene therapy to promote regeneration in Charcot-Marie-Tooth disease. Brain Res. 2019 Oct 24;  146533.

                


                  Waldrop M, Amornvit J, Pierson CR, Boue DR, Sahenk Z. A Novel De Novo Heterozygous SCN4a Mutation Causing Congenital Myopathy, Myotonia and Multiple Congenital Anomalies. J Neuromuscul Dis. 2019 Oct 8;  

                


                  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.

                


                  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;  

                


                  Kopp F, Elguindy MM, Yalvac ME, Zhang H, Chen B, Gillett FA, Lee S, Sivakumar S, Yu H, Xie Y, Mishra P, Sahenk Z, Mendell JT. PUMILIO hyperactivity drives premature aging of Norad-deficient mice. Elife. 2019 Feb 8; 8: 

                


                  Sahenk Z, Yalvac ME, Amornvit J, Arnold WD, Chen L, Shontz KM, Lewis S. Efficacy of exogenous pyruvate in TremblerJ mouse model of Charcot-Marie-Tooth neuropathy. Brain Behav. 2018 Sep 21;  e01118.

                


                  Charleston JS, Schnell FJ, Dworzak J, Donoghue C, Lewis S, Chen L, Young GD, Milici AJ, Voss J, DeAlwis U, Wentworth B, Rodino-Klapac LR, Sahenk Z, Frank D, Mendell JR. Eteplirsen treatment for Duchenne muscular dystrophy: Exon skipping and dystrophin production. Neurology. 2018 Jun 12; 90: e2146-e2154.

                


                  Yalvac ME, Amornvit J, Chen L, Shontz KM, Lewis S, Sahenk Z. AAV1.NT-3 gene therapy increases muscle fiber diameter through activation of mTOR pathway and metabolic remodeling in a CMT mouse model. Gene Ther. 2018 Apr; 25: 129-138.

                


                  Wallace LM, Saad NY, Pyne NK, Fowler AM, Eidahl JO, Domire JS, Griffin DA, Herman AC, Sahenk Z, Rodino-Klapac LR, Harper SQ. Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD. Mol Ther Methods Clin Dev. 2018 Mar 16; 8: 121-130.

View More Publications

Education

Date of Appointment at Nationwide Children’s Hospital: 06/30/2005

Board Certifications

Neurology

Fellowship

Ohio State University Wexner Medical Center

Date Completed: 06/30/1978

Residency

Ohio State University Wexner Medical Center

Date Completed: 06/30/1975

Residency

Hacettepe University

Date Completed: 09/30/1972

Internship

Hacettepe University

Date Completed: 06/30/1972

Medical School

Hacettepe University

Date Completed: 07/31/1972

Professional Experience

2005 - Present The Research Institute at Nationwide Children’s Hospital, Neuromuscular Program, Neuromuscular Pathology, Professor of Neurology and Pediatrics, Director1999 - Present Department of Neurology, Ohio State University College of Medicine, Professor of Neurology1999 - 2005 Division of Neuromuscular Disease, Department of Neurology, Ohio State University Hospitals, Director of the Clinical and Experimental Neuromuscular Disease Laboratories1983 - 1999 Department of Neurology, Ohio State University College of Medicine, Associate Professor or Neurology1986 - 1987 Bio-Architectonics Center, Case Western Reserve University, Visiting Scientist1978 - 1983 Department of Neurology, Ohio State University College of Medicine, Assistant Professor

Contact Information

Pediatrics

Call us at: (614) 722-2203

Fax us at: (614) 355-5247

                    Neurology700 Children's DrColumbus, OH 43205 (map)

Contact Information

Call us at: (614) 722-2203

Fax us at: (614) 355-5247

PediatricsNeurology700 Children’s DrColumbus, OH 43205 (map)

Learn more about Zarife Sahenk

Biography

Zarife Sahenk, MD, PhD, is an attending neurologist at Nationwide Children’s, Director of Clinical and Experimental Neuromuscular Pathology at The Research Institute at Nationwide Children’s and Professor of Pediatrics, Pathology and Neurology at The Ohio State University College of Medicine. Her laboratory plays an integral part in the preclinical studies and the gene therapy clinical trials that take place in the Center for Gene Therapy at Nationwide Children’s Hospital. She has provided valuable analysis of muscle derived from the first clinical gene trials for Duchenne muscular dystrophy, Limb Girdle muscular dystrophy, and follistatin gene therapy for Becker muscular dystrophy, and inclusion body myositis. A primary goal of her research program is to gain a detailed understanding of the anatomical and molecular basis of nerve degeneration and impaired regeneration in hereditary peripheral neuropathies. Particular emphasis is placed on the role of trophic factors secreted by Schwann cells in regeneration-associated myelination. The role of neurotrophin-3 (NT-3) in nerve regeneration in animal models of Charcot-Marie-Tooth (CMT) is currently being investigated and a gene therapy paradigm is being developed using AAV1.NT-3. In addition, a novel translational therapeutic approach that combines vasoactive intestinal peptide (VIP), a lentivirus vector and bone marrow derived dendritic cells for the treatment of chronic inflammatory demyelinating polyradiculoneuropathy is currently being developed in her laboratory. Dr. Sahenk is named among the Best Doctors in America.

Patient Care

Locations

Westerville Surgery Center

Main Campus of Nationwide Children’s Hospital

Academic and Clinical Areas

Neurology

Physician Team

Neuromuscular Disorders

Physician Team

Center for Gene Therapy

Principal Investigator

Neurology Residency

Faculty

Primary Department

Pediatrics

Primary Section

Neurology

Awards, Honors & Organizations

Listed, Best Doctors in America

Research

Lab(s)

Center for Gene Therapy

A primary goal of Dr. Sahenk’s research program is to gain a detailed understanding of the anatomical and molecular basis of hereditary peripheral neuropathies. Particular emphasis is placed on the role of trophic factors secreted by Schwann cells in regeneration-associated myelination. The role of neurotrophin-3 in nerve regeneration in patients and animal models of Charcot-Marie-Tooth (CMT) is currently being investigated. These studies are relevant to understanding the mechanism of axonal loss and impaired regeneration in CMT disorders and developing rational therapies to promote nerve repair. Publications

                  Pozsgai E, Griffin D, Potter R, Sahenk Z, Lehman K, Rodino-Klapac LR, Mendell JR. Unmet needs and evolving treatment for limb girdle muscular dystrophies. Neurodegener Dis Manag. 2021 Oct; 11: 411-429.

                


                  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.

                


                  Jiang B, Sahenk Z, Satoskar A, Freimer M, Ayoub I. Vasculitic neuropathy associated with IgG4-related kidney disease: A case report and literature review. Clin Nephrol. 2021 Sep; 96: 175-179.

                


                  Ozes B, Myers M, Moss K, Mckinney J, Ridgley A, Chen L, Bai S, Abrams CK, Freidin MM, Mendell JR, Sahenk Z. AAV1.NT-3 gene therapy for X-linked Charcot-Marie-Tooth neuropathy type 1. Gene Ther. 2021 Feb 4;  

                


                  Ozes B, Moss K, Myers M, Ridgley A, Chen L, Murrey D, Sahenk Z. AAV1.NT-3 gene therapy in a CMT2D model: phenotypic improvements in GarsP278KY/+ mice. Brain Commun. 2021; 3: fcab252.

                


                  Mendell JR, Sahenk Z, Lehman K, Nease C, Lowes LP, Miller NF, Iammarino MA, Alfano LN, Nicholl A, Al-Zaidy S, Lewis S, Church K, Shell R, Cripe LH, Potter RA, Griffin DA, Pozsgai E, Dugar A, Hogan M, Rodino-Klapac LR. Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial. JAMA Neurol. 2020 Jun 15;  

                


                  Sahenk Z, Ak BO. Gene therapy to promote regeneration in Charcot-Marie-Tooth disease. Brain Res. 2019 Oct 24;  146533.

                


                  Waldrop M, Amornvit J, Pierson CR, Boue DR, Sahenk Z. A Novel De Novo Heterozygous SCN4a Mutation Causing Congenital Myopathy, Myotonia and Multiple Congenital Anomalies. J Neuromuscul Dis. 2019 Oct 8;  

                


                  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.

                


                  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;  

                


                  Kopp F, Elguindy MM, Yalvac ME, Zhang H, Chen B, Gillett FA, Lee S, Sivakumar S, Yu H, Xie Y, Mishra P, Sahenk Z, Mendell JT. PUMILIO hyperactivity drives premature aging of Norad-deficient mice. Elife. 2019 Feb 8; 8: 

                


                  Sahenk Z, Yalvac ME, Amornvit J, Arnold WD, Chen L, Shontz KM, Lewis S. Efficacy of exogenous pyruvate in TremblerJ mouse model of Charcot-Marie-Tooth neuropathy. Brain Behav. 2018 Sep 21;  e01118.

                


                  Charleston JS, Schnell FJ, Dworzak J, Donoghue C, Lewis S, Chen L, Young GD, Milici AJ, Voss J, DeAlwis U, Wentworth B, Rodino-Klapac LR, Sahenk Z, Frank D, Mendell JR. Eteplirsen treatment for Duchenne muscular dystrophy: Exon skipping and dystrophin production. Neurology. 2018 Jun 12; 90: e2146-e2154.

                


                  Yalvac ME, Amornvit J, Chen L, Shontz KM, Lewis S, Sahenk Z. AAV1.NT-3 gene therapy increases muscle fiber diameter through activation of mTOR pathway and metabolic remodeling in a CMT mouse model. Gene Ther. 2018 Apr; 25: 129-138.

                


                  Wallace LM, Saad NY, Pyne NK, Fowler AM, Eidahl JO, Domire JS, Griffin DA, Herman AC, Sahenk Z, Rodino-Klapac LR, Harper SQ. Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD. Mol Ther Methods Clin Dev. 2018 Mar 16; 8: 121-130.

View More Publications

Education

Date of Appointment at Nationwide Children’s Hospital: 06/30/2005

Board Certifications

Neurology

Fellowship

Ohio State University Wexner Medical Center

Date Completed: 06/30/1978

Residency

Ohio State University Wexner Medical Center

Date Completed: 06/30/1975

Residency

Hacettepe University

Date Completed: 09/30/1972

Internship

Hacettepe University

Date Completed: 06/30/1972

Medical School

Hacettepe University

Date Completed: 07/31/1972

Professional Experience

2005 - Present The Research Institute at Nationwide Children’s Hospital, Neuromuscular Program, Neuromuscular Pathology, Professor of Neurology and Pediatrics, Director1999 - Present Department of Neurology, Ohio State University College of Medicine, Professor of Neurology1999 - 2005 Division of Neuromuscular Disease, Department of Neurology, Ohio State University Hospitals, Director of the Clinical and Experimental Neuromuscular Disease Laboratories1983 - 1999 Department of Neurology, Ohio State University College of Medicine, Associate Professor or Neurology1986 - 1987 Bio-Architectonics Center, Case Western Reserve University, Visiting Scientist1978 - 1983 Department of Neurology, Ohio State University College of Medicine, Assistant Professor

Contact Information

Pediatrics

Call us at: (614) 722-2203

Fax us at: (614) 355-5247

                    Neurology700 Children's DrColumbus, OH 43205 (map)

Contact Information

Call us at: (614) 722-2203

Fax us at: (614) 355-5247

PediatricsNeurology700 Children’s DrColumbus, OH 43205 (map)

Learn more about Zarife Sahenk

Contact Information

• Call us at:
• (614) 722-2203
• Fax us at:
• (614) 355-5247
• PediatricsNeurology700 Children’s DrColumbus, OH 43205 (map)

Learn more about Zarife Sahenk

Biography

Zarife Sahenk, MD, PhD, is an attending neurologist at Nationwide Children’s, Director of Clinical and Experimental Neuromuscular Pathology at The Research Institute at Nationwide Children’s and Professor of Pediatrics, Pathology and Neurology at The Ohio State University College of Medicine. Her laboratory plays an integral part in the preclinical studies and the gene therapy clinical trials that take place in the Center for Gene Therapy at Nationwide Children’s Hospital. She has provided valuable analysis of muscle derived from the first clinical gene trials for Duchenne muscular dystrophy, Limb Girdle muscular dystrophy, and follistatin gene therapy for Becker muscular dystrophy, and inclusion body myositis. A primary goal of her research program is to gain a detailed understanding of the anatomical and molecular basis of nerve degeneration and impaired regeneration in hereditary peripheral neuropathies. Particular emphasis is placed on the role of trophic factors secreted by Schwann cells in regeneration-associated myelination. The role of neurotrophin-3 (NT-3) in nerve regeneration in animal models of Charcot-Marie-Tooth (CMT) is currently being investigated and a gene therapy paradigm is being developed using AAV1.NT-3. In addition, a novel translational therapeutic approach that combines vasoactive intestinal peptide (VIP), a lentivirus vector and bone marrow derived dendritic cells for the treatment of chronic inflammatory demyelinating polyradiculoneuropathy is currently being developed in her laboratory. Dr. Sahenk is named among the Best Doctors in America.

Biography

Zarife Sahenk, MD, PhD, is an attending neurologist at Nationwide Children’s, Director of Clinical and Experimental Neuromuscular Pathology at The Research Institute at Nationwide Children’s and Professor of Pediatrics, Pathology and Neurology at The Ohio State University College of Medicine. Her laboratory plays an integral part in the preclinical studies and the gene therapy clinical trials that take place in the Center for Gene Therapy at Nationwide Children’s Hospital. She has provided valuable analysis of muscle derived from the first clinical gene trials for Duchenne muscular dystrophy, Limb Girdle muscular dystrophy, and follistatin gene therapy for Becker muscular dystrophy, and inclusion body myositis. A primary goal of her research program is to gain a detailed understanding of the anatomical and molecular basis of nerve degeneration and impaired regeneration in hereditary peripheral neuropathies. Particular emphasis is placed on the role of trophic factors secreted by Schwann cells in regeneration-associated myelination. The role of neurotrophin-3 (NT-3) in nerve regeneration in animal models of Charcot-Marie-Tooth (CMT) is currently being investigated and a gene therapy paradigm is being developed using AAV1.NT-3. In addition, a novel translational therapeutic approach that combines vasoactive intestinal peptide (VIP), a lentivirus vector and bone marrow derived dendritic cells for the treatment of chronic inflammatory demyelinating polyradiculoneuropathy is currently being developed in her laboratory. Dr. Sahenk is named among the Best Doctors in America.

Biography

Zarife Sahenk, MD, PhD, is an attending neurologist at Nationwide Children’s, Director of Clinical and Experimental Neuromuscular Pathology at The Research Institute at Nationwide Children’s and Professor of Pediatrics, Pathology and Neurology at The Ohio State University College of Medicine. Her laboratory plays an integral part in the preclinical studies and the gene therapy clinical trials that take place in the Center for Gene Therapy at Nationwide Children’s Hospital. She has provided valuable analysis of muscle derived from the first clinical gene trials for Duchenne muscular dystrophy, Limb Girdle muscular dystrophy, and follistatin gene therapy for Becker muscular dystrophy, and inclusion body myositis. A primary goal of her research program is to gain a detailed understanding of the anatomical and molecular basis of nerve degeneration and impaired regeneration in hereditary peripheral neuropathies. Particular emphasis is placed on the role of trophic factors secreted by Schwann cells in regeneration-associated myelination. The role of neurotrophin-3 (NT-3) in nerve regeneration in animal models of Charcot-Marie-Tooth (CMT) is currently being investigated and a gene therapy paradigm is being developed using AAV1.NT-3. In addition, a novel translational therapeutic approach that combines vasoactive intestinal peptide (VIP), a lentivirus vector and bone marrow derived dendritic cells for the treatment of chronic inflammatory demyelinating polyradiculoneuropathy is currently being developed in her laboratory. Dr. Sahenk is named among the Best Doctors in America.

Zarife Sahenk, MD, PhD, is an attending neurologist at Nationwide Children’s, Director of Clinical and Experimental Neuromuscular Pathology at The Research Institute at Nationwide Children’s and Professor of Pediatrics, Pathology and Neurology at The Ohio State University College of Medicine. Her laboratory plays an integral part in the preclinical studies and the gene therapy clinical trials that take place in the Center for Gene Therapy at Nationwide Children’s Hospital. She has provided valuable analysis of muscle derived from the first clinical gene trials for Duchenne muscular dystrophy, Limb Girdle muscular dystrophy, and follistatin gene therapy for Becker muscular dystrophy, and inclusion body myositis. A primary goal of her research program is to gain a detailed understanding of the anatomical and molecular basis of nerve degeneration and impaired regeneration in hereditary peripheral neuropathies. Particular emphasis is placed on the role of trophic factors secreted by Schwann cells in regeneration-associated myelination. The role of neurotrophin-3 (NT-3) in nerve regeneration in animal models of Charcot-Marie-Tooth (CMT) is currently being investigated and a gene therapy paradigm is being developed using AAV1.NT-3. In addition, a novel translational therapeutic approach that combines vasoactive intestinal peptide (VIP), a lentivirus vector and bone marrow derived dendritic cells for the treatment of chronic inflammatory demyelinating polyradiculoneuropathy is currently being developed in her laboratory. Dr. Sahenk is named among the Best Doctors in America.

Zarife Sahenk, MD, PhD, is an attending neurologist at Nationwide Children’s, Director of Clinical and Experimental Neuromuscular Pathology at The Research Institute at Nationwide Children’s and Professor of Pediatrics, Pathology and Neurology at The Ohio State University College of Medicine.

Her laboratory plays an integral part in the preclinical studies and the gene therapy clinical trials that take place in the Center for Gene Therapy at Nationwide Children’s Hospital. She has provided valuable analysis of muscle derived from the first clinical gene trials for Duchenne muscular dystrophy, Limb Girdle muscular dystrophy, and follistatin gene therapy for Becker muscular dystrophy, and inclusion body myositis. A primary goal of her research program is to gain a detailed understanding of the anatomical and molecular basis of nerve degeneration and impaired regeneration in hereditary peripheral neuropathies. Particular emphasis is placed on the role of trophic factors secreted by Schwann cells in regeneration-associated myelination. The role of neurotrophin-3 (NT-3) in nerve regeneration in animal models of Charcot-Marie-Tooth (CMT) is currently being investigated and a gene therapy paradigm is being developed using AAV1.NT-3. In addition, a novel translational therapeutic approach that combines vasoactive intestinal peptide (VIP), a lentivirus vector and bone marrow derived dendritic cells for the treatment of chronic inflammatory demyelinating polyradiculoneuropathy is currently being developed in her laboratory.

Dr. Sahenk is named among the Best Doctors in America.

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

Neurology

Physician Team

Neuromuscular Disorders

Physician Team

Center for Gene Therapy

Principal Investigator

Neurology Residency

Faculty

Primary Department

Pediatrics

Primary Section

Neurology

Academic and Clinical Areas

Neurology

Physician Team

Neuromuscular Disorders

Physician Team

Center for Gene Therapy

Principal Investigator

Neurology Residency

Faculty

Primary Department

Pediatrics

Primary Section

Neurology

Academic and Clinical Areas

Neurology

Physician Team

Neuromuscular Disorders

Physician Team

Center for Gene Therapy

Principal Investigator

Neurology Residency

Faculty

Primary Department

Pediatrics

Primary Section

Neurology

Neurology

Physician Team

Neuromuscular Disorders

Physician Team

Center for Gene Therapy

Principal Investigator

Neurology Residency

Faculty

Primary Department

Pediatrics

Primary Section

Neurology

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

Awards, Honors & Organizations

Listed, Best Doctors in America

Awards, Honors & Organizations

Listed, Best Doctors in America

Awards, Honors & Organizations

Listed, Best Doctors in America

Listed, Best Doctors in America

• Listed, Best Doctors in America

Research

Lab(s)

Center for Gene Therapy

A primary goal of Dr. Sahenk’s research program is to gain a detailed understanding of the anatomical and molecular basis of hereditary peripheral neuropathies. Particular emphasis is placed on the role of trophic factors secreted by Schwann cells in regeneration-associated myelination. The role of neurotrophin-3 in nerve regeneration in patients and animal models of Charcot-Marie-Tooth (CMT) is currently being investigated. These studies are relevant to understanding the mechanism of axonal loss and impaired regeneration in CMT disorders and developing rational therapies to promote nerve repair. Publications

                  Pozsgai E, Griffin D, Potter R, Sahenk Z, Lehman K, Rodino-Klapac LR, Mendell JR. Unmet needs and evolving treatment for limb girdle muscular dystrophies. Neurodegener Dis Manag. 2021 Oct; 11: 411-429.

                


                  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.

                


                  Jiang B, Sahenk Z, Satoskar A, Freimer M, Ayoub I. Vasculitic neuropathy associated with IgG4-related kidney disease: A case report and literature review. Clin Nephrol. 2021 Sep; 96: 175-179.

                


                  Ozes B, Myers M, Moss K, Mckinney J, Ridgley A, Chen L, Bai S, Abrams CK, Freidin MM, Mendell JR, Sahenk Z. AAV1.NT-3 gene therapy for X-linked Charcot-Marie-Tooth neuropathy type 1. Gene Ther. 2021 Feb 4;  

                


                  Ozes B, Moss K, Myers M, Ridgley A, Chen L, Murrey D, Sahenk Z. AAV1.NT-3 gene therapy in a CMT2D model: phenotypic improvements in GarsP278KY/+ mice. Brain Commun. 2021; 3: fcab252.

                


                  Mendell JR, Sahenk Z, Lehman K, Nease C, Lowes LP, Miller NF, Iammarino MA, Alfano LN, Nicholl A, Al-Zaidy S, Lewis S, Church K, Shell R, Cripe LH, Potter RA, Griffin DA, Pozsgai E, Dugar A, Hogan M, Rodino-Klapac LR. Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial. JAMA Neurol. 2020 Jun 15;  

                


                  Sahenk Z, Ak BO. Gene therapy to promote regeneration in Charcot-Marie-Tooth disease. Brain Res. 2019 Oct 24;  146533.

                


                  Waldrop M, Amornvit J, Pierson CR, Boue DR, Sahenk Z. A Novel De Novo Heterozygous SCN4a Mutation Causing Congenital Myopathy, Myotonia and Multiple Congenital Anomalies. J Neuromuscul Dis. 2019 Oct 8;  

                


                  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.

                


                  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;  

                


                  Kopp F, Elguindy MM, Yalvac ME, Zhang H, Chen B, Gillett FA, Lee S, Sivakumar S, Yu H, Xie Y, Mishra P, Sahenk Z, Mendell JT. PUMILIO hyperactivity drives premature aging of Norad-deficient mice. Elife. 2019 Feb 8; 8: 

                


                  Sahenk Z, Yalvac ME, Amornvit J, Arnold WD, Chen L, Shontz KM, Lewis S. Efficacy of exogenous pyruvate in TremblerJ mouse model of Charcot-Marie-Tooth neuropathy. Brain Behav. 2018 Sep 21;  e01118.

                


                  Charleston JS, Schnell FJ, Dworzak J, Donoghue C, Lewis S, Chen L, Young GD, Milici AJ, Voss J, DeAlwis U, Wentworth B, Rodino-Klapac LR, Sahenk Z, Frank D, Mendell JR. Eteplirsen treatment for Duchenne muscular dystrophy: Exon skipping and dystrophin production. Neurology. 2018 Jun 12; 90: e2146-e2154.

                


                  Yalvac ME, Amornvit J, Chen L, Shontz KM, Lewis S, Sahenk Z. AAV1.NT-3 gene therapy increases muscle fiber diameter through activation of mTOR pathway and metabolic remodeling in a CMT mouse model. Gene Ther. 2018 Apr; 25: 129-138.

                


                  Wallace LM, Saad NY, Pyne NK, Fowler AM, Eidahl JO, Domire JS, Griffin DA, Herman AC, Sahenk Z, Rodino-Klapac LR, Harper SQ. Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD. Mol Ther Methods Clin Dev. 2018 Mar 16; 8: 121-130.

View More Publications

Research

Lab(s)

Center for Gene Therapy

A primary goal of Dr. Sahenk’s research program is to gain a detailed understanding of the anatomical and molecular basis of hereditary peripheral neuropathies. Particular emphasis is placed on the role of trophic factors secreted by Schwann cells in regeneration-associated myelination. The role of neurotrophin-3 in nerve regeneration in patients and animal models of Charcot-Marie-Tooth (CMT) is currently being investigated. These studies are relevant to understanding the mechanism of axonal loss and impaired regeneration in CMT disorders and developing rational therapies to promote nerve repair. Publications

                  Pozsgai E, Griffin D, Potter R, Sahenk Z, Lehman K, Rodino-Klapac LR, Mendell JR. Unmet needs and evolving treatment for limb girdle muscular dystrophies. Neurodegener Dis Manag. 2021 Oct; 11: 411-429.

                


                  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.

                


                  Jiang B, Sahenk Z, Satoskar A, Freimer M, Ayoub I. Vasculitic neuropathy associated with IgG4-related kidney disease: A case report and literature review. Clin Nephrol. 2021 Sep; 96: 175-179.

                


                  Ozes B, Myers M, Moss K, Mckinney J, Ridgley A, Chen L, Bai S, Abrams CK, Freidin MM, Mendell JR, Sahenk Z. AAV1.NT-3 gene therapy for X-linked Charcot-Marie-Tooth neuropathy type 1. Gene Ther. 2021 Feb 4;  

                


                  Ozes B, Moss K, Myers M, Ridgley A, Chen L, Murrey D, Sahenk Z. AAV1.NT-3 gene therapy in a CMT2D model: phenotypic improvements in GarsP278KY/+ mice. Brain Commun. 2021; 3: fcab252.

                


                  Mendell JR, Sahenk Z, Lehman K, Nease C, Lowes LP, Miller NF, Iammarino MA, Alfano LN, Nicholl A, Al-Zaidy S, Lewis S, Church K, Shell R, Cripe LH, Potter RA, Griffin DA, Pozsgai E, Dugar A, Hogan M, Rodino-Klapac LR. Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial. JAMA Neurol. 2020 Jun 15;  

                


                  Sahenk Z, Ak BO. Gene therapy to promote regeneration in Charcot-Marie-Tooth disease. Brain Res. 2019 Oct 24;  146533.

                


                  Waldrop M, Amornvit J, Pierson CR, Boue DR, Sahenk Z. A Novel De Novo Heterozygous SCN4a Mutation Causing Congenital Myopathy, Myotonia and Multiple Congenital Anomalies. J Neuromuscul Dis. 2019 Oct 8;  

                


                  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.

                


                  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;  

                


                  Kopp F, Elguindy MM, Yalvac ME, Zhang H, Chen B, Gillett FA, Lee S, Sivakumar S, Yu H, Xie Y, Mishra P, Sahenk Z, Mendell JT. PUMILIO hyperactivity drives premature aging of Norad-deficient mice. Elife. 2019 Feb 8; 8: 

                


                  Sahenk Z, Yalvac ME, Amornvit J, Arnold WD, Chen L, Shontz KM, Lewis S. Efficacy of exogenous pyruvate in TremblerJ mouse model of Charcot-Marie-Tooth neuropathy. Brain Behav. 2018 Sep 21;  e01118.

                


                  Charleston JS, Schnell FJ, Dworzak J, Donoghue C, Lewis S, Chen L, Young GD, Milici AJ, Voss J, DeAlwis U, Wentworth B, Rodino-Klapac LR, Sahenk Z, Frank D, Mendell JR. Eteplirsen treatment for Duchenne muscular dystrophy: Exon skipping and dystrophin production. Neurology. 2018 Jun 12; 90: e2146-e2154.

                


                  Yalvac ME, Amornvit J, Chen L, Shontz KM, Lewis S, Sahenk Z. AAV1.NT-3 gene therapy increases muscle fiber diameter through activation of mTOR pathway and metabolic remodeling in a CMT mouse model. Gene Ther. 2018 Apr; 25: 129-138.

                


                  Wallace LM, Saad NY, Pyne NK, Fowler AM, Eidahl JO, Domire JS, Griffin DA, Herman AC, Sahenk Z, Rodino-Klapac LR, Harper SQ. Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD. Mol Ther Methods Clin Dev. 2018 Mar 16; 8: 121-130.

View More Publications

Research

Lab(s)

Center for Gene Therapy

A primary goal of Dr. Sahenk’s research program is to gain a detailed understanding of the anatomical and molecular basis of hereditary peripheral neuropathies. Particular emphasis is placed on the role of trophic factors secreted by Schwann cells in regeneration-associated myelination. The role of neurotrophin-3 in nerve regeneration in patients and animal models of Charcot-Marie-Tooth (CMT) is currently being investigated. These studies are relevant to understanding the mechanism of axonal loss and impaired regeneration in CMT disorders and developing rational therapies to promote nerve repair. Publications

                  Pozsgai E, Griffin D, Potter R, Sahenk Z, Lehman K, Rodino-Klapac LR, Mendell JR. Unmet needs and evolving treatment for limb girdle muscular dystrophies. Neurodegener Dis Manag. 2021 Oct; 11: 411-429.

                


                  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.

                


                  Jiang B, Sahenk Z, Satoskar A, Freimer M, Ayoub I. Vasculitic neuropathy associated with IgG4-related kidney disease: A case report and literature review. Clin Nephrol. 2021 Sep; 96: 175-179.

                


                  Ozes B, Myers M, Moss K, Mckinney J, Ridgley A, Chen L, Bai S, Abrams CK, Freidin MM, Mendell JR, Sahenk Z. AAV1.NT-3 gene therapy for X-linked Charcot-Marie-Tooth neuropathy type 1. Gene Ther. 2021 Feb 4;  

                


                  Ozes B, Moss K, Myers M, Ridgley A, Chen L, Murrey D, Sahenk Z. AAV1.NT-3 gene therapy in a CMT2D model: phenotypic improvements in GarsP278KY/+ mice. Brain Commun. 2021; 3: fcab252.

                


                  Mendell JR, Sahenk Z, Lehman K, Nease C, Lowes LP, Miller NF, Iammarino MA, Alfano LN, Nicholl A, Al-Zaidy S, Lewis S, Church K, Shell R, Cripe LH, Potter RA, Griffin DA, Pozsgai E, Dugar A, Hogan M, Rodino-Klapac LR. Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial. JAMA Neurol. 2020 Jun 15;  

                


                  Sahenk Z, Ak BO. Gene therapy to promote regeneration in Charcot-Marie-Tooth disease. Brain Res. 2019 Oct 24;  146533.

                


                  Waldrop M, Amornvit J, Pierson CR, Boue DR, Sahenk Z. A Novel De Novo Heterozygous SCN4a Mutation Causing Congenital Myopathy, Myotonia and Multiple Congenital Anomalies. J Neuromuscul Dis. 2019 Oct 8;  

                


                  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.

                


                  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;  

                


                  Kopp F, Elguindy MM, Yalvac ME, Zhang H, Chen B, Gillett FA, Lee S, Sivakumar S, Yu H, Xie Y, Mishra P, Sahenk Z, Mendell JT. PUMILIO hyperactivity drives premature aging of Norad-deficient mice. Elife. 2019 Feb 8; 8: 

                


                  Sahenk Z, Yalvac ME, Amornvit J, Arnold WD, Chen L, Shontz KM, Lewis S. Efficacy of exogenous pyruvate in TremblerJ mouse model of Charcot-Marie-Tooth neuropathy. Brain Behav. 2018 Sep 21;  e01118.

                


                  Charleston JS, Schnell FJ, Dworzak J, Donoghue C, Lewis S, Chen L, Young GD, Milici AJ, Voss J, DeAlwis U, Wentworth B, Rodino-Klapac LR, Sahenk Z, Frank D, Mendell JR. Eteplirsen treatment for Duchenne muscular dystrophy: Exon skipping and dystrophin production. Neurology. 2018 Jun 12; 90: e2146-e2154.

                


                  Yalvac ME, Amornvit J, Chen L, Shontz KM, Lewis S, Sahenk Z. AAV1.NT-3 gene therapy increases muscle fiber diameter through activation of mTOR pathway and metabolic remodeling in a CMT mouse model. Gene Ther. 2018 Apr; 25: 129-138.

                


                  Wallace LM, Saad NY, Pyne NK, Fowler AM, Eidahl JO, Domire JS, Griffin DA, Herman AC, Sahenk Z, Rodino-Klapac LR, Harper SQ. Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD. Mol Ther Methods Clin Dev. 2018 Mar 16; 8: 121-130.

View More Publications

Lab(s)

Center for Gene Therapy

A primary goal of Dr. Sahenk’s research program is to gain a detailed understanding of the anatomical and molecular basis of hereditary peripheral neuropathies. Particular emphasis is placed on the role of trophic factors secreted by Schwann cells in regeneration-associated myelination. The role of neurotrophin-3 in nerve regeneration in patients and animal models of Charcot-Marie-Tooth (CMT) is currently being investigated. These studies are relevant to understanding the mechanism of axonal loss and impaired regeneration in CMT disorders and developing rational therapies to promote nerve repair. Publications

                  Pozsgai E, Griffin D, Potter R, Sahenk Z, Lehman K, Rodino-Klapac LR, Mendell JR. Unmet needs and evolving treatment for limb girdle muscular dystrophies. Neurodegener Dis Manag. 2021 Oct; 11: 411-429.

                


                  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.

                


                  Jiang B, Sahenk Z, Satoskar A, Freimer M, Ayoub I. Vasculitic neuropathy associated with IgG4-related kidney disease: A case report and literature review. Clin Nephrol. 2021 Sep; 96: 175-179.

                


                  Ozes B, Myers M, Moss K, Mckinney J, Ridgley A, Chen L, Bai S, Abrams CK, Freidin MM, Mendell JR, Sahenk Z. AAV1.NT-3 gene therapy for X-linked Charcot-Marie-Tooth neuropathy type 1. Gene Ther. 2021 Feb 4;  

                


                  Ozes B, Moss K, Myers M, Ridgley A, Chen L, Murrey D, Sahenk Z. AAV1.NT-3 gene therapy in a CMT2D model: phenotypic improvements in GarsP278KY/+ mice. Brain Commun. 2021; 3: fcab252.

                


                  Mendell JR, Sahenk Z, Lehman K, Nease C, Lowes LP, Miller NF, Iammarino MA, Alfano LN, Nicholl A, Al-Zaidy S, Lewis S, Church K, Shell R, Cripe LH, Potter RA, Griffin DA, Pozsgai E, Dugar A, Hogan M, Rodino-Klapac LR. Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial. JAMA Neurol. 2020 Jun 15;  

                


                  Sahenk Z, Ak BO. Gene therapy to promote regeneration in Charcot-Marie-Tooth disease. Brain Res. 2019 Oct 24;  146533.

                


                  Waldrop M, Amornvit J, Pierson CR, Boue DR, Sahenk Z. A Novel De Novo Heterozygous SCN4a Mutation Causing Congenital Myopathy, Myotonia and Multiple Congenital Anomalies. J Neuromuscul Dis. 2019 Oct 8;  

                


                  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.

                


                  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;  

                


                  Kopp F, Elguindy MM, Yalvac ME, Zhang H, Chen B, Gillett FA, Lee S, Sivakumar S, Yu H, Xie Y, Mishra P, Sahenk Z, Mendell JT. PUMILIO hyperactivity drives premature aging of Norad-deficient mice. Elife. 2019 Feb 8; 8: 

                


                  Sahenk Z, Yalvac ME, Amornvit J, Arnold WD, Chen L, Shontz KM, Lewis S. Efficacy of exogenous pyruvate in TremblerJ mouse model of Charcot-Marie-Tooth neuropathy. Brain Behav. 2018 Sep 21;  e01118.

                


                  Charleston JS, Schnell FJ, Dworzak J, Donoghue C, Lewis S, Chen L, Young GD, Milici AJ, Voss J, DeAlwis U, Wentworth B, Rodino-Klapac LR, Sahenk Z, Frank D, Mendell JR. Eteplirsen treatment for Duchenne muscular dystrophy: Exon skipping and dystrophin production. Neurology. 2018 Jun 12; 90: e2146-e2154.

                


                  Yalvac ME, Amornvit J, Chen L, Shontz KM, Lewis S, Sahenk Z. AAV1.NT-3 gene therapy increases muscle fiber diameter through activation of mTOR pathway and metabolic remodeling in a CMT mouse model. Gene Ther. 2018 Apr; 25: 129-138.

                


                  Wallace LM, Saad NY, Pyne NK, Fowler AM, Eidahl JO, Domire JS, Griffin DA, Herman AC, Sahenk Z, Rodino-Klapac LR, Harper SQ. Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD. Mol Ther Methods Clin Dev. 2018 Mar 16; 8: 121-130.

View More Publications

Lab(s)

Center for Gene Therapy

A primary goal of Dr. Sahenk’s research program is to gain a detailed understanding of the anatomical and molecular basis of hereditary peripheral neuropathies. Particular emphasis is placed on the role of trophic factors secreted by Schwann cells in regeneration-associated myelination. The role of neurotrophin-3 in nerve regeneration in patients and animal models of Charcot-Marie-Tooth (CMT) is currently being investigated. These studies are relevant to understanding the mechanism of axonal loss and impaired regeneration in CMT disorders and developing rational therapies to promote nerve repair.

Lab(s)

Center for Gene Therapy

• Center for Gene Therapy

                  Pozsgai E, Griffin D, Potter R, Sahenk Z, Lehman K, Rodino-Klapac LR, Mendell JR. Unmet needs and evolving treatment for limb girdle muscular dystrophies. Neurodegener Dis Manag. 2021 Oct; 11: 411-429.
  
  
  
                  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.
  
  
  
                  Jiang B, Sahenk Z, Satoskar A, Freimer M, Ayoub I. Vasculitic neuropathy associated with IgG4-related kidney disease: A case report and literature review. Clin Nephrol. 2021 Sep; 96: 175-179.
  
  
  
                  Ozes B, Myers M, Moss K, Mckinney J, Ridgley A, Chen L, Bai S, Abrams CK, Freidin MM, Mendell JR, Sahenk Z. AAV1.NT-3 gene therapy for X-linked Charcot-Marie-Tooth neuropathy type 1. Gene Ther. 2021 Feb 4;  
  
  
  
                  Ozes B, Moss K, Myers M, Ridgley A, Chen L, Murrey D, Sahenk Z. AAV1.NT-3 gene therapy in a CMT2D model: phenotypic improvements in GarsP278KY/+ mice. Brain Commun. 2021; 3: fcab252.
  
  
  
                  Mendell JR, Sahenk Z, Lehman K, Nease C, Lowes LP, Miller NF, Iammarino MA, Alfano LN, Nicholl A, Al-Zaidy S, Lewis S, Church K, Shell R, Cripe LH, Potter RA, Griffin DA, Pozsgai E, Dugar A, Hogan M, Rodino-Klapac LR. Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial. JAMA Neurol. 2020 Jun 15;  
  
  
  
                  Sahenk Z, Ak BO. Gene therapy to promote regeneration in Charcot-Marie-Tooth disease. Brain Res. 2019 Oct 24;  146533.
  
  
  
                  Waldrop M, Amornvit J, Pierson CR, Boue DR, Sahenk Z. A Novel De Novo Heterozygous SCN4a Mutation Causing Congenital Myopathy, Myotonia and Multiple Congenital Anomalies. J Neuromuscul Dis. 2019 Oct 8;  
  
  
  
                  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.
  
  
  
                  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;  
  
  
  
                  Kopp F, Elguindy MM, Yalvac ME, Zhang H, Chen B, Gillett FA, Lee S, Sivakumar S, Yu H, Xie Y, Mishra P, Sahenk Z, Mendell JT. PUMILIO hyperactivity drives premature aging of Norad-deficient mice. Elife. 2019 Feb 8; 8: 
  
  
  
                  Sahenk Z, Yalvac ME, Amornvit J, Arnold WD, Chen L, Shontz KM, Lewis S. Efficacy of exogenous pyruvate in TremblerJ mouse model of Charcot-Marie-Tooth neuropathy. Brain Behav. 2018 Sep 21;  e01118.
  
  
  
                  Charleston JS, Schnell FJ, Dworzak J, Donoghue C, Lewis S, Chen L, Young GD, Milici AJ, Voss J, DeAlwis U, Wentworth B, Rodino-Klapac LR, Sahenk Z, Frank D, Mendell JR. Eteplirsen treatment for Duchenne muscular dystrophy: Exon skipping and dystrophin production. Neurology. 2018 Jun 12; 90: e2146-e2154.
  
  
  
                  Yalvac ME, Amornvit J, Chen L, Shontz KM, Lewis S, Sahenk Z. AAV1.NT-3 gene therapy increases muscle fiber diameter through activation of mTOR pathway and metabolic remodeling in a CMT mouse model. Gene Ther. 2018 Apr; 25: 129-138.
  
  
  
                  Wallace LM, Saad NY, Pyne NK, Fowler AM, Eidahl JO, Domire JS, Griffin DA, Herman AC, Sahenk Z, Rodino-Klapac LR, Harper SQ. Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD. Mol Ther Methods Clin Dev. 2018 Mar 16; 8: 121-130.
  
  

View More Publications

• Pozsgai E, Griffin D, Potter R, Sahenk Z, Lehman K, Rodino-Klapac LR, Mendell JR. Unmet needs and evolving treatment for limb girdle muscular dystrophies. Neurodegener Dis Manag. 2021 Oct; 11: 411-429.
• 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.
• Jiang B, Sahenk Z, Satoskar A, Freimer M, Ayoub I. Vasculitic neuropathy associated with IgG4-related kidney disease: A case report and literature review. Clin Nephrol. 2021 Sep; 96: 175-179.
• Ozes B, Myers M, Moss K, Mckinney J, Ridgley A, Chen L, Bai S, Abrams CK, Freidin MM, Mendell JR, Sahenk Z. AAV1.NT-3 gene therapy for X-linked Charcot-Marie-Tooth neuropathy type 1. Gene Ther. 2021 Feb 4;
• Ozes B, Moss K, Myers M, Ridgley A, Chen L, Murrey D, Sahenk Z. AAV1.NT-3 gene therapy in a CMT2D model: phenotypic improvements in GarsP278KY/+ mice. Brain Commun. 2021; 3: fcab252.
• Mendell JR, Sahenk Z, Lehman K, Nease C, Lowes LP, Miller NF, Iammarino MA, Alfano LN, Nicholl A, Al-Zaidy S, Lewis S, Church K, Shell R, Cripe LH, Potter RA, Griffin DA, Pozsgai E, Dugar A, Hogan M, Rodino-Klapac LR. Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial. JAMA Neurol. 2020 Jun 15;
• Sahenk Z, Ak BO. Gene therapy to promote regeneration in Charcot-Marie-Tooth disease. Brain Res. 2019 Oct 24; 146533.
• Waldrop M, Amornvit J, Pierson CR, Boue DR, Sahenk Z. A Novel De Novo Heterozygous SCN4a Mutation Causing Congenital Myopathy, Myotonia and Multiple Congenital Anomalies. J Neuromuscul Dis. 2019 Oct 8;
• 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.
• 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;
• Kopp F, Elguindy MM, Yalvac ME, Zhang H, Chen B, Gillett FA, Lee S, Sivakumar S, Yu H, Xie Y, Mishra P, Sahenk Z, Mendell JT. PUMILIO hyperactivity drives premature aging of Norad-deficient mice. Elife. 2019 Feb 8; 8:
• Sahenk Z, Yalvac ME, Amornvit J, Arnold WD, Chen L, Shontz KM, Lewis S. Efficacy of exogenous pyruvate in TremblerJ mouse model of Charcot-Marie-Tooth neuropathy. Brain Behav. 2018 Sep 21; e01118.
• Charleston JS, Schnell FJ, Dworzak J, Donoghue C, Lewis S, Chen L, Young GD, Milici AJ, Voss J, DeAlwis U, Wentworth B, Rodino-Klapac LR, Sahenk Z, Frank D, Mendell JR. Eteplirsen treatment for Duchenne muscular dystrophy: Exon skipping and dystrophin production. Neurology. 2018 Jun 12; 90: e2146-e2154.
• Yalvac ME, Amornvit J, Chen L, Shontz KM, Lewis S, Sahenk Z. AAV1.NT-3 gene therapy increases muscle fiber diameter through activation of mTOR pathway and metabolic remodeling in a CMT mouse model. Gene Ther. 2018 Apr; 25: 129-138.
• Wallace LM, Saad NY, Pyne NK, Fowler AM, Eidahl JO, Domire JS, Griffin DA, Herman AC, Sahenk Z, Rodino-Klapac LR, Harper SQ. Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD. Mol Ther Methods Clin Dev. 2018 Mar 16; 8: 121-130.

Education

Date of Appointment at Nationwide Children’s Hospital: 06/30/2005

Board Certifications

Neurology

Fellowship

Ohio State University Wexner Medical Center

Date Completed: 06/30/1978

Residency

Ohio State University Wexner Medical Center

Date Completed: 06/30/1975

Residency

Hacettepe University

Date Completed: 09/30/1972

Internship

Hacettepe University

Date Completed: 06/30/1972

Medical School

Hacettepe University

Date Completed: 07/31/1972

Education

Date of Appointment at Nationwide Children’s Hospital: 06/30/2005

Board Certifications

Neurology

Fellowship

Ohio State University Wexner Medical Center

Date Completed: 06/30/1978

Residency

Ohio State University Wexner Medical Center

Date Completed: 06/30/1975

Residency

Hacettepe University

Date Completed: 09/30/1972

Internship

Hacettepe University

Date Completed: 06/30/1972

Medical School

Hacettepe University

Date Completed: 07/31/1972

Education

Date of Appointment at Nationwide Children’s Hospital: 06/30/2005

Board Certifications

Neurology

Fellowship

Ohio State University Wexner Medical Center

Date Completed: 06/30/1978

Residency

Ohio State University Wexner Medical Center

Date Completed: 06/30/1975

Residency

Hacettepe University

Date Completed: 09/30/1972

Internship

Hacettepe University

Date Completed: 06/30/1972

Medical School

Hacettepe University

Date Completed: 07/31/1972

Date of Appointment at Nationwide Children’s Hospital: 06/30/2005

Board Certifications

Neurology

Fellowship

Ohio State University Wexner Medical Center

Date Completed: 06/30/1978

Residency

Ohio State University Wexner Medical Center

Date Completed: 06/30/1975

Residency

Hacettepe University

Date Completed: 09/30/1972

Internship

Hacettepe University

Date Completed: 06/30/1972

Medical School

Hacettepe University

Date Completed: 07/31/1972

Date of Appointment at Nationwide Children’s Hospital: 06/30/2005

Board Certifications

Neurology

Fellowship

Ohio State University Wexner Medical Center

Date Completed: 06/30/1978

Residency

Ohio State University Wexner Medical Center

Date Completed: 06/30/1975

Residency

Hacettepe University

Date Completed: 09/30/1972

Internship

Hacettepe University

Date Completed: 06/30/1972

Medical School

Hacettepe University

Date Completed: 07/31/1972

• Neurology

Professional Experience

2005 - Present The Research Institute at Nationwide Children’s Hospital, Neuromuscular Program, Neuromuscular Pathology, Professor of Neurology and Pediatrics, Director1999 - Present Department of Neurology, Ohio State University College of Medicine, Professor of Neurology1999 - 2005 Division of Neuromuscular Disease, Department of Neurology, Ohio State University Hospitals, Director of the Clinical and Experimental Neuromuscular Disease Laboratories1983 - 1999 Department of Neurology, Ohio State University College of Medicine, Associate Professor or Neurology1986 - 1987 Bio-Architectonics Center, Case Western Reserve University, Visiting Scientist1978 - 1983 Department of Neurology, Ohio State University College of Medicine, Assistant Professor

Professional Experience

2005 - Present The Research Institute at Nationwide Children’s Hospital, Neuromuscular Program, Neuromuscular Pathology, Professor of Neurology and Pediatrics, Director1999 - Present Department of Neurology, Ohio State University College of Medicine, Professor of Neurology1999 - 2005 Division of Neuromuscular Disease, Department of Neurology, Ohio State University Hospitals, Director of the Clinical and Experimental Neuromuscular Disease Laboratories1983 - 1999 Department of Neurology, Ohio State University College of Medicine, Associate Professor or Neurology1986 - 1987 Bio-Architectonics Center, Case Western Reserve University, Visiting Scientist1978 - 1983 Department of Neurology, Ohio State University College of Medicine, Assistant Professor

Professional Experience

2005 - Present The Research Institute at Nationwide Children’s Hospital, Neuromuscular Program, Neuromuscular Pathology, Professor of Neurology and Pediatrics, Director1999 - Present Department of Neurology, Ohio State University College of Medicine, Professor of Neurology1999 - 2005 Division of Neuromuscular Disease, Department of Neurology, Ohio State University Hospitals, Director of the Clinical and Experimental Neuromuscular Disease Laboratories1983 - 1999 Department of Neurology, Ohio State University College of Medicine, Associate Professor or Neurology1986 - 1987 Bio-Architectonics Center, Case Western Reserve University, Visiting Scientist1978 - 1983 Department of Neurology, Ohio State University College of Medicine, Assistant Professor

2005 - Present The Research Institute at Nationwide Children’s Hospital, Neuromuscular Program, Neuromuscular Pathology, Professor of Neurology and Pediatrics, Director1999 - Present Department of Neurology, Ohio State University College of Medicine, Professor of Neurology1999 - 2005 Division of Neuromuscular Disease, Department of Neurology, Ohio State University Hospitals, Director of the Clinical and Experimental Neuromuscular Disease Laboratories1983 - 1999 Department of Neurology, Ohio State University College of Medicine, Associate Professor or Neurology1986 - 1987 Bio-Architectonics Center, Case Western Reserve University, Visiting Scientist1978 - 1983 Department of Neurology, Ohio State University College of Medicine, Assistant Professor

2005 - Present The Research Institute at Nationwide Children’s Hospital, Neuromuscular Program, Neuromuscular Pathology, Professor of Neurology and Pediatrics, Director

Contact Information

Pediatrics

Call us at: (614) 722-2203

Fax us at: (614) 355-5247

                    Neurology700 Children's DrColumbus, OH 43205 (map)

Contact Information

Pediatrics

Call us at: (614) 722-2203

Fax us at: (614) 355-5247

                    Neurology700 Children's DrColumbus, OH 43205 (map)

Contact Information

Pediatrics

Call us at: (614) 722-2203

Fax us at: (614) 355-5247

                    Neurology700 Children's DrColumbus, OH 43205 (map)

Pediatrics

Call us at: (614) 722-2203

Fax us at: (614) 355-5247

                    Neurology700 Children's DrColumbus, OH 43205 (map)

Call us at: (614) 722-2203

Fax us at: (614) 355-5247

                    Neurology700 Children's DrColumbus, OH 43205 (map)

Call us at: (614) 722-2203

Fax us at: (614) 355-5247

                    Neurology700 Children's DrColumbus, OH 43205 (map)

• Call us at:
• (614) 722-2203
• Fax us at:
• (614) 355-5247
• Neurology700 Children’s DrColumbus, OH 43205 (map)