Contact Information
Call us at: (614) 722-3535
Fax us at: (614) 722-3273
PediatricsCenter for Gene Therapy700 Children’s Drive Rm WA3021Columbus, OH 43205 (map)
Learn more about Stephen G. Kaler
Biography
Stephen G. Kaler, MD, MPH, is a professor of Pediatrics and Genetics and a physician-scientist in the Center for Gene Therapy, Abigail Wexner Research Institute at Nationwide Children’s Hospital. He is triple-certified (Clinical, Biochemical, and Molecular Genetics) by the American Board of Medical Genetics and considered an authority on Menkes disease, a rare inherited disorder of copper metabolism, and its variants. Dr. Kaler earned his medical degree from the University of Rochester, Rochester, NY, and completed an internship in Internal Medicine at Brigham & Women’s Hospital, Boston, MA. He completed residency training in Pediatrics (Loyola University Medical Center, Tufts-New England Medical Center) and fellowship in Medical Genetics at the National Institutes of Health (NIH). He then served as Director of Biochemical and Molecular Genetics at Children’s National Medical Center in Washington, DC and earned an MPH (international health track) from George Washington University School of Public Health before returning to NIH as a Clinical Director and tenured Senior Investigator. He joined the Center for Gene Therapy at Nationwide Children’s Hospital in Fall 2019.
Academic and Clinical Areas
Center for Gene Therapy
Principal Investigator
Genetic and Genomic Medicine
Physician Team
Medical Genetics Residency
Faculty
Primary Department
Pediatrics
Primary Section
Genetic and Genomic Medicine
Awards, Honors & Organizations
Achievement Medal, US Public Health Service Citation, US Public Health Service Commendation Medal, US Public Health Service Certificate of Recognition, NIH Distinguished Mentor Award Program Physician Researcher of the Year, US Office of the Surgeon General Award for Distinguished Service: Ebola Clinical Research Response Team, United States Secretary of Health and Human Services Elected Member, Society for Pediatric Research Elected Member, Association of American Physicians
Research
Lab(s)
Center for Gene Therapy
The Kaler Laboratory is committed to dissecting the mechanisms and pathophysiology of inherited neurometabolic, motor neuron, and copper transport diseases and using the knowledge to improve health through rational remedies, including gene therapy. Patients and families affected by these conditions provide the impetus for scientific inquiry in the Laboratory. In addition to molecular genetics, the Laboratory employs preclinical models, cellular and biochemical approaches, and conducts clinical trials. Adeno-associated virus (AAV)-mediated gene therapy for Menkes disease, choroid plexus–targeted gene therapy for Alpha-mannosidosis, studies of motor neuron degeneration mediated by p97/valosin-containing protein (p97/VCP), and delineation of inherited disorders of ATP7A-related copper transport represent current main directions.
View My Publications
Publications
Sharma P, Reichert M, Lu Y, Markello TC, Adams DR, Steinbach PJ, Fuqua BK, Parisi X, Kaler SG, Vulpe CD, Anderson GJ, Gahl WA, Malicdan MCV. Biallelic HEPHL1 variants impair ferroxidase activity and cause an abnormal hair phenotype. PLoS Genet. 2019 May; 15: e1008143.
PREVAIL III Study Group., Sneller MC, Reilly C, Badio M, Bishop RJ, Eghrari AO, Moses SJ, Johnson KL, Gayedyu-Dennis D, Hensley LE, Higgs ES, Nath A, Tuznik K, Varughese J, Jensen KS, Dighero-Kemp B, Neaton JD, Lane HC, Fallah MP. A Longitudinal Study of Ebola Sequelae in Liberia. N Engl J Med. 2019 Mar 7; 380: 924-934.
Haddad MR, Choi EY, Zerfas PM, Yi L, Martinelli D, Sullivan P, Goldstein DS, Centeno JA, Brinster LR, Ralle M, Kaler SG. Cerebrospinal Fluid-Directed rAAV9-rsATP7A Plus Subcutaneous Copper Histidinate Advance Survival and Outcomes in a Menkes Disease Mouse Model. Mol Ther Methods Clin Dev. 2018 Sep 21; 10: 165-178.
Yi L, Kaler SG. Interaction between the AAA ATPase p97/VCP and a concealed UBX domain in the copper transporter ATP7A is associated with motor neuron degeneration. J Biol Chem. 2018 May 18; 293: 7606-7617.
Kennedy SB, Bolay F, Kieh M, Grandits G, Badio M, Ballou R, Eckes R, Feinberg M, Follmann D, Grund B, Gupta S, Hensley L, Higgs E, Janosko K, Johnson M, Kateh F, Logue J, Marchand J, Monath T, Nason M, Nyenswah T, Roman F, Stavale E, Wolfson J, Neaton JD, Lane HC, PREVAIL I Study Group.. Phase 2 Placebo-Controlled Trial of Two Vaccines to Prevent Ebola in Liberia. N Engl J Med. 2017 Oct 12; 377: 1438-1447.
Fallah MP, Skrip LA, Dahn BT, Nyenswah TG, Flumo H, Glayweon M, Lorseh TL, Kaler SG, Higgs ES, Galvani AP. Pregnancy outcomes in Liberian women who conceived after recovery from Ebola virus disease. Lancet Glob Health. 2016 Oct; 4: e678-9.
Kaler SG. Microbial peptide de-coppers mitochondria: implications for Wilson disease. J Clin Invest. 2016 Jul 1; 126: 2412-4.
Yi L, Kaler SG. Direct interactions of adaptor protein complexes 1 and 2 with the copper transporter ATP7A mediate its anterograde and retrograde trafficking. Hum Mol Genet. 2015 May 1; 24: 2411-25.
Bandmann O, Weiss KH, Kaler SG. Wilson's disease and other neurological copper disorders. Lancet Neurol. 2015 Jan; 14: 103-13.
View More Publications
Education
Date of Appointment at Nationwide Children’s Hospital: 09/27/2019
Board Certifications
Clinical Biochemical/Molecular Genetics Clinical Genetics and Genomics
Fellowship
National Institutes of Health
Date Completed: 06/30/1989
Residency
Tufts New England Medical Center
Date Completed: 06/30/1988
Residency
Loyola University Medical Center
Date Completed: 06/30/1987
Internship
Brigham and Women’s Hospital
Date Completed: 06/30/1985
Medical School
University of Rochester School of Medicine/Dentist
Date Completed: 05/19/1984
Professional Experience
2015 - 2019 NIH, Head, Metals Biology/Molecular Medicine Affinity Group, Intramural Research Program2013 - 2019 NIH, Chair, Trans-NIH Gene Therapy Consortium. Intramural Research Program2001 - 2010 NICHD, NIH, Clinical Director, Intramural Research Program2000 - 2001 National Institutes of Health, Deputy Associate Director for Disease Prevention, Office of the Director1999 - 2000 Children’s Hospital National Medical Center, Director, Biochemical and Molecular Genetics; Department of Laboratory Medicine, Department of Pathology1999 - 2000 Children’s Hospital National Medical Center, Vice Chair, Division of Metabolism1995 - 2000 George Washington University School of Medicine, Associate Professor of Pediatrics and Pathology, Division of Medical Genetics, Department of Pediatrics
Contact Information
Pediatrics
Call us at: (614) 722-3535
Fax us at: (614) 722-3273
Center for Gene Therapy700 Children's Drive Rm WA3021Columbus, OH 43205 (map)
Contact Information
Call us at: (614) 722-3535
Fax us at: (614) 722-3273
PediatricsCenter for Gene Therapy700 Children’s Drive Rm WA3021Columbus, OH 43205 (map)
Learn more about Stephen G. Kaler
Biography
Stephen G. Kaler, MD, MPH, is a professor of Pediatrics and Genetics and a physician-scientist in the Center for Gene Therapy, Abigail Wexner Research Institute at Nationwide Children’s Hospital. He is triple-certified (Clinical, Biochemical, and Molecular Genetics) by the American Board of Medical Genetics and considered an authority on Menkes disease, a rare inherited disorder of copper metabolism, and its variants. Dr. Kaler earned his medical degree from the University of Rochester, Rochester, NY, and completed an internship in Internal Medicine at Brigham & Women’s Hospital, Boston, MA. He completed residency training in Pediatrics (Loyola University Medical Center, Tufts-New England Medical Center) and fellowship in Medical Genetics at the National Institutes of Health (NIH). He then served as Director of Biochemical and Molecular Genetics at Children’s National Medical Center in Washington, DC and earned an MPH (international health track) from George Washington University School of Public Health before returning to NIH as a Clinical Director and tenured Senior Investigator. He joined the Center for Gene Therapy at Nationwide Children’s Hospital in Fall 2019.
Academic and Clinical Areas
Center for Gene Therapy
Principal Investigator
Genetic and Genomic Medicine
Physician Team
Medical Genetics Residency
Faculty
Primary Department
Pediatrics
Primary Section
Genetic and Genomic Medicine
Awards, Honors & Organizations
Achievement Medal, US Public Health Service Citation, US Public Health Service Commendation Medal, US Public Health Service Certificate of Recognition, NIH Distinguished Mentor Award Program Physician Researcher of the Year, US Office of the Surgeon General Award for Distinguished Service: Ebola Clinical Research Response Team, United States Secretary of Health and Human Services Elected Member, Society for Pediatric Research Elected Member, Association of American Physicians
Research
Lab(s)
Center for Gene Therapy
The Kaler Laboratory is committed to dissecting the mechanisms and pathophysiology of inherited neurometabolic, motor neuron, and copper transport diseases and using the knowledge to improve health through rational remedies, including gene therapy. Patients and families affected by these conditions provide the impetus for scientific inquiry in the Laboratory. In addition to molecular genetics, the Laboratory employs preclinical models, cellular and biochemical approaches, and conducts clinical trials. Adeno-associated virus (AAV)-mediated gene therapy for Menkes disease, choroid plexus–targeted gene therapy for Alpha-mannosidosis, studies of motor neuron degeneration mediated by p97/valosin-containing protein (p97/VCP), and delineation of inherited disorders of ATP7A-related copper transport represent current main directions.
View My Publications
Publications
Sharma P, Reichert M, Lu Y, Markello TC, Adams DR, Steinbach PJ, Fuqua BK, Parisi X, Kaler SG, Vulpe CD, Anderson GJ, Gahl WA, Malicdan MCV. Biallelic HEPHL1 variants impair ferroxidase activity and cause an abnormal hair phenotype. PLoS Genet. 2019 May; 15: e1008143.
PREVAIL III Study Group., Sneller MC, Reilly C, Badio M, Bishop RJ, Eghrari AO, Moses SJ, Johnson KL, Gayedyu-Dennis D, Hensley LE, Higgs ES, Nath A, Tuznik K, Varughese J, Jensen KS, Dighero-Kemp B, Neaton JD, Lane HC, Fallah MP. A Longitudinal Study of Ebola Sequelae in Liberia. N Engl J Med. 2019 Mar 7; 380: 924-934.
Haddad MR, Choi EY, Zerfas PM, Yi L, Martinelli D, Sullivan P, Goldstein DS, Centeno JA, Brinster LR, Ralle M, Kaler SG. Cerebrospinal Fluid-Directed rAAV9-rsATP7A Plus Subcutaneous Copper Histidinate Advance Survival and Outcomes in a Menkes Disease Mouse Model. Mol Ther Methods Clin Dev. 2018 Sep 21; 10: 165-178.
Yi L, Kaler SG. Interaction between the AAA ATPase p97/VCP and a concealed UBX domain in the copper transporter ATP7A is associated with motor neuron degeneration. J Biol Chem. 2018 May 18; 293: 7606-7617.
Kennedy SB, Bolay F, Kieh M, Grandits G, Badio M, Ballou R, Eckes R, Feinberg M, Follmann D, Grund B, Gupta S, Hensley L, Higgs E, Janosko K, Johnson M, Kateh F, Logue J, Marchand J, Monath T, Nason M, Nyenswah T, Roman F, Stavale E, Wolfson J, Neaton JD, Lane HC, PREVAIL I Study Group.. Phase 2 Placebo-Controlled Trial of Two Vaccines to Prevent Ebola in Liberia. N Engl J Med. 2017 Oct 12; 377: 1438-1447.
Fallah MP, Skrip LA, Dahn BT, Nyenswah TG, Flumo H, Glayweon M, Lorseh TL, Kaler SG, Higgs ES, Galvani AP. Pregnancy outcomes in Liberian women who conceived after recovery from Ebola virus disease. Lancet Glob Health. 2016 Oct; 4: e678-9.
Kaler SG. Microbial peptide de-coppers mitochondria: implications for Wilson disease. J Clin Invest. 2016 Jul 1; 126: 2412-4.
Yi L, Kaler SG. Direct interactions of adaptor protein complexes 1 and 2 with the copper transporter ATP7A mediate its anterograde and retrograde trafficking. Hum Mol Genet. 2015 May 1; 24: 2411-25.
Bandmann O, Weiss KH, Kaler SG. Wilson's disease and other neurological copper disorders. Lancet Neurol. 2015 Jan; 14: 103-13.
View More Publications
Education
Date of Appointment at Nationwide Children’s Hospital: 09/27/2019
Board Certifications
Clinical Biochemical/Molecular Genetics Clinical Genetics and Genomics
Fellowship
National Institutes of Health
Date Completed: 06/30/1989
Residency
Tufts New England Medical Center
Date Completed: 06/30/1988
Residency
Loyola University Medical Center
Date Completed: 06/30/1987
Internship
Brigham and Women’s Hospital
Date Completed: 06/30/1985
Medical School
University of Rochester School of Medicine/Dentist
Date Completed: 05/19/1984
Professional Experience
2015 - 2019 NIH, Head, Metals Biology/Molecular Medicine Affinity Group, Intramural Research Program2013 - 2019 NIH, Chair, Trans-NIH Gene Therapy Consortium. Intramural Research Program2001 - 2010 NICHD, NIH, Clinical Director, Intramural Research Program2000 - 2001 National Institutes of Health, Deputy Associate Director for Disease Prevention, Office of the Director1999 - 2000 Children’s Hospital National Medical Center, Director, Biochemical and Molecular Genetics; Department of Laboratory Medicine, Department of Pathology1999 - 2000 Children’s Hospital National Medical Center, Vice Chair, Division of Metabolism1995 - 2000 George Washington University School of Medicine, Associate Professor of Pediatrics and Pathology, Division of Medical Genetics, Department of Pediatrics
Contact Information
Pediatrics
Call us at: (614) 722-3535
Fax us at: (614) 722-3273
Center for Gene Therapy700 Children's Drive Rm WA3021Columbus, OH 43205 (map)
Contact Information
Call us at: (614) 722-3535
Fax us at: (614) 722-3273
PediatricsCenter for Gene Therapy700 Children’s Drive Rm WA3021Columbus, OH 43205 (map)
Learn more about Stephen G. Kaler
Contact Information
- Call us at:
- (614) 722-3535
- Fax us at:
- (614) 722-3273
- PediatricsCenter for Gene Therapy700 Children’s Drive Rm WA3021Columbus, OH 43205 (map)
Learn more about Stephen G. Kaler
Biography
Stephen G. Kaler, MD, MPH, is a professor of Pediatrics and Genetics and a physician-scientist in the Center for Gene Therapy, Abigail Wexner Research Institute at Nationwide Children’s Hospital. He is triple-certified (Clinical, Biochemical, and Molecular Genetics) by the American Board of Medical Genetics and considered an authority on Menkes disease, a rare inherited disorder of copper metabolism, and its variants. Dr. Kaler earned his medical degree from the University of Rochester, Rochester, NY, and completed an internship in Internal Medicine at Brigham & Women’s Hospital, Boston, MA. He completed residency training in Pediatrics (Loyola University Medical Center, Tufts-New England Medical Center) and fellowship in Medical Genetics at the National Institutes of Health (NIH). He then served as Director of Biochemical and Molecular Genetics at Children’s National Medical Center in Washington, DC and earned an MPH (international health track) from George Washington University School of Public Health before returning to NIH as a Clinical Director and tenured Senior Investigator. He joined the Center for Gene Therapy at Nationwide Children’s Hospital in Fall 2019.
Biography
Stephen G. Kaler, MD, MPH, is a professor of Pediatrics and Genetics and a physician-scientist in the Center for Gene Therapy, Abigail Wexner Research Institute at Nationwide Children’s Hospital. He is triple-certified (Clinical, Biochemical, and Molecular Genetics) by the American Board of Medical Genetics and considered an authority on Menkes disease, a rare inherited disorder of copper metabolism, and its variants. Dr. Kaler earned his medical degree from the University of Rochester, Rochester, NY, and completed an internship in Internal Medicine at Brigham & Women’s Hospital, Boston, MA. He completed residency training in Pediatrics (Loyola University Medical Center, Tufts-New England Medical Center) and fellowship in Medical Genetics at the National Institutes of Health (NIH). He then served as Director of Biochemical and Molecular Genetics at Children’s National Medical Center in Washington, DC and earned an MPH (international health track) from George Washington University School of Public Health before returning to NIH as a Clinical Director and tenured Senior Investigator. He joined the Center for Gene Therapy at Nationwide Children’s Hospital in Fall 2019.
Biography
Stephen G. Kaler, MD, MPH, is a professor of Pediatrics and Genetics and a physician-scientist in the Center for Gene Therapy, Abigail Wexner Research Institute at Nationwide Children’s Hospital. He is triple-certified (Clinical, Biochemical, and Molecular Genetics) by the American Board of Medical Genetics and considered an authority on Menkes disease, a rare inherited disorder of copper metabolism, and its variants. Dr. Kaler earned his medical degree from the University of Rochester, Rochester, NY, and completed an internship in Internal Medicine at Brigham & Women’s Hospital, Boston, MA. He completed residency training in Pediatrics (Loyola University Medical Center, Tufts-New England Medical Center) and fellowship in Medical Genetics at the National Institutes of Health (NIH). He then served as Director of Biochemical and Molecular Genetics at Children’s National Medical Center in Washington, DC and earned an MPH (international health track) from George Washington University School of Public Health before returning to NIH as a Clinical Director and tenured Senior Investigator. He joined the Center for Gene Therapy at Nationwide Children’s Hospital in Fall 2019.
Stephen G. Kaler, MD, MPH, is a professor of Pediatrics and Genetics and a physician-scientist in the Center for Gene Therapy, Abigail Wexner Research Institute at Nationwide Children’s Hospital. He is triple-certified (Clinical, Biochemical, and Molecular Genetics) by the American Board of Medical Genetics and considered an authority on Menkes disease, a rare inherited disorder of copper metabolism, and its variants. Dr. Kaler earned his medical degree from the University of Rochester, Rochester, NY, and completed an internship in Internal Medicine at Brigham & Women’s Hospital, Boston, MA. He completed residency training in Pediatrics (Loyola University Medical Center, Tufts-New England Medical Center) and fellowship in Medical Genetics at the National Institutes of Health (NIH). He then served as Director of Biochemical and Molecular Genetics at Children’s National Medical Center in Washington, DC and earned an MPH (international health track) from George Washington University School of Public Health before returning to NIH as a Clinical Director and tenured Senior Investigator. He joined the Center for Gene Therapy at Nationwide Children’s Hospital in Fall 2019.
Stephen G. Kaler, MD, MPH, is a professor of Pediatrics and Genetics and a physician-scientist in the Center for Gene Therapy, Abigail Wexner Research Institute at Nationwide Children’s Hospital. He is triple-certified (Clinical, Biochemical, and Molecular Genetics) by the American Board of Medical Genetics and considered an authority on Menkes disease, a rare inherited disorder of copper metabolism, and its variants.
Dr. Kaler earned his medical degree from the University of Rochester, Rochester, NY, and completed an internship in Internal Medicine at Brigham & Women’s Hospital, Boston, MA. He completed residency training in Pediatrics (Loyola University Medical Center, Tufts-New England Medical Center) and fellowship in Medical Genetics at the National Institutes of Health (NIH). He then served as Director of Biochemical and Molecular Genetics at Children’s National Medical Center in Washington, DC and earned an MPH (international health track) from George Washington University School of Public Health before returning to NIH as a Clinical Director and tenured Senior Investigator. He joined the Center for Gene Therapy at Nationwide Children’s Hospital in Fall 2019.
Academic and Clinical Areas
Center for Gene Therapy
Principal Investigator
Genetic and Genomic Medicine
Physician Team
Medical Genetics Residency
Faculty
Primary Department
Pediatrics
Primary Section
Genetic and Genomic Medicine
Academic and Clinical Areas
Center for Gene Therapy
Principal Investigator
Genetic and Genomic Medicine
Physician Team
Medical Genetics Residency
Faculty
Primary Department
Pediatrics
Primary Section
Genetic and Genomic Medicine
Academic and Clinical Areas
Center for Gene Therapy
Principal Investigator
Genetic and Genomic Medicine
Physician Team
Medical Genetics Residency
Faculty
Primary Department
Pediatrics
Primary Section
Genetic and Genomic Medicine
Center for Gene Therapy
Principal Investigator
Genetic and Genomic Medicine
Physician Team
Medical Genetics Residency
Faculty
Primary Department
Pediatrics
Primary Section
Genetic and Genomic Medicine
- Center for Gene Therapy
- Principal Investigator
- Genetic and Genomic Medicine
- Physician Team
- Medical Genetics Residency
- Faculty
- Primary Department
- Pediatrics
- Primary Section
- Genetic and Genomic Medicine
Awards, Honors & Organizations
Achievement Medal, US Public Health Service Citation, US Public Health Service Commendation Medal, US Public Health Service Certificate of Recognition, NIH Distinguished Mentor Award Program Physician Researcher of the Year, US Office of the Surgeon General Award for Distinguished Service: Ebola Clinical Research Response Team, United States Secretary of Health and Human Services Elected Member, Society for Pediatric Research Elected Member, Association of American Physicians
Awards, Honors & Organizations
Achievement Medal, US Public Health Service Citation, US Public Health Service Commendation Medal, US Public Health Service Certificate of Recognition, NIH Distinguished Mentor Award Program Physician Researcher of the Year, US Office of the Surgeon General Award for Distinguished Service: Ebola Clinical Research Response Team, United States Secretary of Health and Human Services Elected Member, Society for Pediatric Research Elected Member, Association of American Physicians
Awards, Honors & Organizations
Achievement Medal, US Public Health Service Citation, US Public Health Service Commendation Medal, US Public Health Service Certificate of Recognition, NIH Distinguished Mentor Award Program Physician Researcher of the Year, US Office of the Surgeon General Award for Distinguished Service: Ebola Clinical Research Response Team, United States Secretary of Health and Human Services Elected Member, Society for Pediatric Research Elected Member, Association of American Physicians
Achievement Medal, US Public Health Service Citation, US Public Health Service Commendation Medal, US Public Health Service Certificate of Recognition, NIH Distinguished Mentor Award Program Physician Researcher of the Year, US Office of the Surgeon General Award for Distinguished Service: Ebola Clinical Research Response Team, United States Secretary of Health and Human Services Elected Member, Society for Pediatric Research Elected Member, Association of American Physicians
- Achievement Medal, US Public Health Service
- Citation, US Public Health Service
- Commendation Medal, US Public Health Service
- Certificate of Recognition, NIH Distinguished Mentor Award Program
- Physician Researcher of the Year, US Office of the Surgeon General
- Award for Distinguished Service: Ebola Clinical Research Response Team, United States Secretary of Health and Human Services
- Elected Member, Society for Pediatric Research
- Elected Member, Association of American Physicians
Research
Lab(s)
Center for Gene Therapy
The Kaler Laboratory is committed to dissecting the mechanisms and pathophysiology of inherited neurometabolic, motor neuron, and copper transport diseases and using the knowledge to improve health through rational remedies, including gene therapy. Patients and families affected by these conditions provide the impetus for scientific inquiry in the Laboratory. In addition to molecular genetics, the Laboratory employs preclinical models, cellular and biochemical approaches, and conducts clinical trials. Adeno-associated virus (AAV)-mediated gene therapy for Menkes disease, choroid plexus–targeted gene therapy for Alpha-mannosidosis, studies of motor neuron degeneration mediated by p97/valosin-containing protein (p97/VCP), and delineation of inherited disorders of ATP7A-related copper transport represent current main directions.
View My Publications
Publications
Sharma P, Reichert M, Lu Y, Markello TC, Adams DR, Steinbach PJ, Fuqua BK, Parisi X, Kaler SG, Vulpe CD, Anderson GJ, Gahl WA, Malicdan MCV. Biallelic HEPHL1 variants impair ferroxidase activity and cause an abnormal hair phenotype. PLoS Genet. 2019 May; 15: e1008143.
PREVAIL III Study Group., Sneller MC, Reilly C, Badio M, Bishop RJ, Eghrari AO, Moses SJ, Johnson KL, Gayedyu-Dennis D, Hensley LE, Higgs ES, Nath A, Tuznik K, Varughese J, Jensen KS, Dighero-Kemp B, Neaton JD, Lane HC, Fallah MP. A Longitudinal Study of Ebola Sequelae in Liberia. N Engl J Med. 2019 Mar 7; 380: 924-934.
Haddad MR, Choi EY, Zerfas PM, Yi L, Martinelli D, Sullivan P, Goldstein DS, Centeno JA, Brinster LR, Ralle M, Kaler SG. Cerebrospinal Fluid-Directed rAAV9-rsATP7A Plus Subcutaneous Copper Histidinate Advance Survival and Outcomes in a Menkes Disease Mouse Model. Mol Ther Methods Clin Dev. 2018 Sep 21; 10: 165-178.
Yi L, Kaler SG. Interaction between the AAA ATPase p97/VCP and a concealed UBX domain in the copper transporter ATP7A is associated with motor neuron degeneration. J Biol Chem. 2018 May 18; 293: 7606-7617.
Kennedy SB, Bolay F, Kieh M, Grandits G, Badio M, Ballou R, Eckes R, Feinberg M, Follmann D, Grund B, Gupta S, Hensley L, Higgs E, Janosko K, Johnson M, Kateh F, Logue J, Marchand J, Monath T, Nason M, Nyenswah T, Roman F, Stavale E, Wolfson J, Neaton JD, Lane HC, PREVAIL I Study Group.. Phase 2 Placebo-Controlled Trial of Two Vaccines to Prevent Ebola in Liberia. N Engl J Med. 2017 Oct 12; 377: 1438-1447.
Fallah MP, Skrip LA, Dahn BT, Nyenswah TG, Flumo H, Glayweon M, Lorseh TL, Kaler SG, Higgs ES, Galvani AP. Pregnancy outcomes in Liberian women who conceived after recovery from Ebola virus disease. Lancet Glob Health. 2016 Oct; 4: e678-9.
Kaler SG. Microbial peptide de-coppers mitochondria: implications for Wilson disease. J Clin Invest. 2016 Jul 1; 126: 2412-4.
Yi L, Kaler SG. Direct interactions of adaptor protein complexes 1 and 2 with the copper transporter ATP7A mediate its anterograde and retrograde trafficking. Hum Mol Genet. 2015 May 1; 24: 2411-25.
Bandmann O, Weiss KH, Kaler SG. Wilson's disease and other neurological copper disorders. Lancet Neurol. 2015 Jan; 14: 103-13.
View More Publications
Research
Lab(s)
Center for Gene Therapy
The Kaler Laboratory is committed to dissecting the mechanisms and pathophysiology of inherited neurometabolic, motor neuron, and copper transport diseases and using the knowledge to improve health through rational remedies, including gene therapy. Patients and families affected by these conditions provide the impetus for scientific inquiry in the Laboratory. In addition to molecular genetics, the Laboratory employs preclinical models, cellular and biochemical approaches, and conducts clinical trials. Adeno-associated virus (AAV)-mediated gene therapy for Menkes disease, choroid plexus–targeted gene therapy for Alpha-mannosidosis, studies of motor neuron degeneration mediated by p97/valosin-containing protein (p97/VCP), and delineation of inherited disorders of ATP7A-related copper transport represent current main directions.
View My Publications
Publications
Sharma P, Reichert M, Lu Y, Markello TC, Adams DR, Steinbach PJ, Fuqua BK, Parisi X, Kaler SG, Vulpe CD, Anderson GJ, Gahl WA, Malicdan MCV. Biallelic HEPHL1 variants impair ferroxidase activity and cause an abnormal hair phenotype. PLoS Genet. 2019 May; 15: e1008143.
PREVAIL III Study Group., Sneller MC, Reilly C, Badio M, Bishop RJ, Eghrari AO, Moses SJ, Johnson KL, Gayedyu-Dennis D, Hensley LE, Higgs ES, Nath A, Tuznik K, Varughese J, Jensen KS, Dighero-Kemp B, Neaton JD, Lane HC, Fallah MP. A Longitudinal Study of Ebola Sequelae in Liberia. N Engl J Med. 2019 Mar 7; 380: 924-934.
Haddad MR, Choi EY, Zerfas PM, Yi L, Martinelli D, Sullivan P, Goldstein DS, Centeno JA, Brinster LR, Ralle M, Kaler SG. Cerebrospinal Fluid-Directed rAAV9-rsATP7A Plus Subcutaneous Copper Histidinate Advance Survival and Outcomes in a Menkes Disease Mouse Model. Mol Ther Methods Clin Dev. 2018 Sep 21; 10: 165-178.
Yi L, Kaler SG. Interaction between the AAA ATPase p97/VCP and a concealed UBX domain in the copper transporter ATP7A is associated with motor neuron degeneration. J Biol Chem. 2018 May 18; 293: 7606-7617.
Kennedy SB, Bolay F, Kieh M, Grandits G, Badio M, Ballou R, Eckes R, Feinberg M, Follmann D, Grund B, Gupta S, Hensley L, Higgs E, Janosko K, Johnson M, Kateh F, Logue J, Marchand J, Monath T, Nason M, Nyenswah T, Roman F, Stavale E, Wolfson J, Neaton JD, Lane HC, PREVAIL I Study Group.. Phase 2 Placebo-Controlled Trial of Two Vaccines to Prevent Ebola in Liberia. N Engl J Med. 2017 Oct 12; 377: 1438-1447.
Fallah MP, Skrip LA, Dahn BT, Nyenswah TG, Flumo H, Glayweon M, Lorseh TL, Kaler SG, Higgs ES, Galvani AP. Pregnancy outcomes in Liberian women who conceived after recovery from Ebola virus disease. Lancet Glob Health. 2016 Oct; 4: e678-9.
Kaler SG. Microbial peptide de-coppers mitochondria: implications for Wilson disease. J Clin Invest. 2016 Jul 1; 126: 2412-4.
Yi L, Kaler SG. Direct interactions of adaptor protein complexes 1 and 2 with the copper transporter ATP7A mediate its anterograde and retrograde trafficking. Hum Mol Genet. 2015 May 1; 24: 2411-25.
Bandmann O, Weiss KH, Kaler SG. Wilson's disease and other neurological copper disorders. Lancet Neurol. 2015 Jan; 14: 103-13.
View More Publications
Research
Lab(s)
Center for Gene Therapy
The Kaler Laboratory is committed to dissecting the mechanisms and pathophysiology of inherited neurometabolic, motor neuron, and copper transport diseases and using the knowledge to improve health through rational remedies, including gene therapy. Patients and families affected by these conditions provide the impetus for scientific inquiry in the Laboratory. In addition to molecular genetics, the Laboratory employs preclinical models, cellular and biochemical approaches, and conducts clinical trials. Adeno-associated virus (AAV)-mediated gene therapy for Menkes disease, choroid plexus–targeted gene therapy for Alpha-mannosidosis, studies of motor neuron degeneration mediated by p97/valosin-containing protein (p97/VCP), and delineation of inherited disorders of ATP7A-related copper transport represent current main directions.
View My Publications
Publications
Sharma P, Reichert M, Lu Y, Markello TC, Adams DR, Steinbach PJ, Fuqua BK, Parisi X, Kaler SG, Vulpe CD, Anderson GJ, Gahl WA, Malicdan MCV. Biallelic HEPHL1 variants impair ferroxidase activity and cause an abnormal hair phenotype. PLoS Genet. 2019 May; 15: e1008143.
PREVAIL III Study Group., Sneller MC, Reilly C, Badio M, Bishop RJ, Eghrari AO, Moses SJ, Johnson KL, Gayedyu-Dennis D, Hensley LE, Higgs ES, Nath A, Tuznik K, Varughese J, Jensen KS, Dighero-Kemp B, Neaton JD, Lane HC, Fallah MP. A Longitudinal Study of Ebola Sequelae in Liberia. N Engl J Med. 2019 Mar 7; 380: 924-934.
Haddad MR, Choi EY, Zerfas PM, Yi L, Martinelli D, Sullivan P, Goldstein DS, Centeno JA, Brinster LR, Ralle M, Kaler SG. Cerebrospinal Fluid-Directed rAAV9-rsATP7A Plus Subcutaneous Copper Histidinate Advance Survival and Outcomes in a Menkes Disease Mouse Model. Mol Ther Methods Clin Dev. 2018 Sep 21; 10: 165-178.
Yi L, Kaler SG. Interaction between the AAA ATPase p97/VCP and a concealed UBX domain in the copper transporter ATP7A is associated with motor neuron degeneration. J Biol Chem. 2018 May 18; 293: 7606-7617.
Kennedy SB, Bolay F, Kieh M, Grandits G, Badio M, Ballou R, Eckes R, Feinberg M, Follmann D, Grund B, Gupta S, Hensley L, Higgs E, Janosko K, Johnson M, Kateh F, Logue J, Marchand J, Monath T, Nason M, Nyenswah T, Roman F, Stavale E, Wolfson J, Neaton JD, Lane HC, PREVAIL I Study Group.. Phase 2 Placebo-Controlled Trial of Two Vaccines to Prevent Ebola in Liberia. N Engl J Med. 2017 Oct 12; 377: 1438-1447.
Fallah MP, Skrip LA, Dahn BT, Nyenswah TG, Flumo H, Glayweon M, Lorseh TL, Kaler SG, Higgs ES, Galvani AP. Pregnancy outcomes in Liberian women who conceived after recovery from Ebola virus disease. Lancet Glob Health. 2016 Oct; 4: e678-9.
Kaler SG. Microbial peptide de-coppers mitochondria: implications for Wilson disease. J Clin Invest. 2016 Jul 1; 126: 2412-4.
Yi L, Kaler SG. Direct interactions of adaptor protein complexes 1 and 2 with the copper transporter ATP7A mediate its anterograde and retrograde trafficking. Hum Mol Genet. 2015 May 1; 24: 2411-25.
Bandmann O, Weiss KH, Kaler SG. Wilson's disease and other neurological copper disorders. Lancet Neurol. 2015 Jan; 14: 103-13.
View More Publications
Lab(s)
Center for Gene Therapy
The Kaler Laboratory is committed to dissecting the mechanisms and pathophysiology of inherited neurometabolic, motor neuron, and copper transport diseases and using the knowledge to improve health through rational remedies, including gene therapy. Patients and families affected by these conditions provide the impetus for scientific inquiry in the Laboratory. In addition to molecular genetics, the Laboratory employs preclinical models, cellular and biochemical approaches, and conducts clinical trials. Adeno-associated virus (AAV)-mediated gene therapy for Menkes disease, choroid plexus–targeted gene therapy for Alpha-mannosidosis, studies of motor neuron degeneration mediated by p97/valosin-containing protein (p97/VCP), and delineation of inherited disorders of ATP7A-related copper transport represent current main directions.
View My Publications
Publications
Sharma P, Reichert M, Lu Y, Markello TC, Adams DR, Steinbach PJ, Fuqua BK, Parisi X, Kaler SG, Vulpe CD, Anderson GJ, Gahl WA, Malicdan MCV. Biallelic HEPHL1 variants impair ferroxidase activity and cause an abnormal hair phenotype. PLoS Genet. 2019 May; 15: e1008143.
PREVAIL III Study Group., Sneller MC, Reilly C, Badio M, Bishop RJ, Eghrari AO, Moses SJ, Johnson KL, Gayedyu-Dennis D, Hensley LE, Higgs ES, Nath A, Tuznik K, Varughese J, Jensen KS, Dighero-Kemp B, Neaton JD, Lane HC, Fallah MP. A Longitudinal Study of Ebola Sequelae in Liberia. N Engl J Med. 2019 Mar 7; 380: 924-934.
Haddad MR, Choi EY, Zerfas PM, Yi L, Martinelli D, Sullivan P, Goldstein DS, Centeno JA, Brinster LR, Ralle M, Kaler SG. Cerebrospinal Fluid-Directed rAAV9-rsATP7A Plus Subcutaneous Copper Histidinate Advance Survival and Outcomes in a Menkes Disease Mouse Model. Mol Ther Methods Clin Dev. 2018 Sep 21; 10: 165-178.
Yi L, Kaler SG. Interaction between the AAA ATPase p97/VCP and a concealed UBX domain in the copper transporter ATP7A is associated with motor neuron degeneration. J Biol Chem. 2018 May 18; 293: 7606-7617.
Kennedy SB, Bolay F, Kieh M, Grandits G, Badio M, Ballou R, Eckes R, Feinberg M, Follmann D, Grund B, Gupta S, Hensley L, Higgs E, Janosko K, Johnson M, Kateh F, Logue J, Marchand J, Monath T, Nason M, Nyenswah T, Roman F, Stavale E, Wolfson J, Neaton JD, Lane HC, PREVAIL I Study Group.. Phase 2 Placebo-Controlled Trial of Two Vaccines to Prevent Ebola in Liberia. N Engl J Med. 2017 Oct 12; 377: 1438-1447.
Fallah MP, Skrip LA, Dahn BT, Nyenswah TG, Flumo H, Glayweon M, Lorseh TL, Kaler SG, Higgs ES, Galvani AP. Pregnancy outcomes in Liberian women who conceived after recovery from Ebola virus disease. Lancet Glob Health. 2016 Oct; 4: e678-9.
Kaler SG. Microbial peptide de-coppers mitochondria: implications for Wilson disease. J Clin Invest. 2016 Jul 1; 126: 2412-4.
Yi L, Kaler SG. Direct interactions of adaptor protein complexes 1 and 2 with the copper transporter ATP7A mediate its anterograde and retrograde trafficking. Hum Mol Genet. 2015 May 1; 24: 2411-25.
Bandmann O, Weiss KH, Kaler SG. Wilson's disease and other neurological copper disorders. Lancet Neurol. 2015 Jan; 14: 103-13.
View More Publications
Lab(s)
Center for Gene Therapy
The Kaler Laboratory is committed to dissecting the mechanisms and pathophysiology of inherited neurometabolic, motor neuron, and copper transport diseases and using the knowledge to improve health through rational remedies, including gene therapy. Patients and families affected by these conditions provide the impetus for scientific inquiry in the Laboratory. In addition to molecular genetics, the Laboratory employs preclinical models, cellular and biochemical approaches, and conducts clinical trials. Adeno-associated virus (AAV)-mediated gene therapy for Menkes disease, choroid plexus–targeted gene therapy for Alpha-mannosidosis, studies of motor neuron degeneration mediated by p97/valosin-containing protein (p97/VCP), and delineation of inherited disorders of ATP7A-related copper transport represent current main directions.
Lab(s)
Center for Gene Therapy
Center for Gene Therapy
View My Publications
Sharma P, Reichert M, Lu Y, Markello TC, Adams DR, Steinbach PJ, Fuqua BK, Parisi X, Kaler SG, Vulpe CD, Anderson GJ, Gahl WA, Malicdan MCV. Biallelic HEPHL1 variants impair ferroxidase activity and cause an abnormal hair phenotype. PLoS Genet. 2019 May; 15: e1008143. PREVAIL III Study Group., Sneller MC, Reilly C, Badio M, Bishop RJ, Eghrari AO, Moses SJ, Johnson KL, Gayedyu-Dennis D, Hensley LE, Higgs ES, Nath A, Tuznik K, Varughese J, Jensen KS, Dighero-Kemp B, Neaton JD, Lane HC, Fallah MP. A Longitudinal Study of Ebola Sequelae in Liberia. N Engl J Med. 2019 Mar 7; 380: 924-934. Haddad MR, Choi EY, Zerfas PM, Yi L, Martinelli D, Sullivan P, Goldstein DS, Centeno JA, Brinster LR, Ralle M, Kaler SG. Cerebrospinal Fluid-Directed rAAV9-rsATP7A Plus Subcutaneous Copper Histidinate Advance Survival and Outcomes in a Menkes Disease Mouse Model. Mol Ther Methods Clin Dev. 2018 Sep 21; 10: 165-178. Yi L, Kaler SG. Interaction between the AAA ATPase p97/VCP and a concealed UBX domain in the copper transporter ATP7A is associated with motor neuron degeneration. J Biol Chem. 2018 May 18; 293: 7606-7617. Kennedy SB, Bolay F, Kieh M, Grandits G, Badio M, Ballou R, Eckes R, Feinberg M, Follmann D, Grund B, Gupta S, Hensley L, Higgs E, Janosko K, Johnson M, Kateh F, Logue J, Marchand J, Monath T, Nason M, Nyenswah T, Roman F, Stavale E, Wolfson J, Neaton JD, Lane HC, PREVAIL I Study Group.. Phase 2 Placebo-Controlled Trial of Two Vaccines to Prevent Ebola in Liberia. N Engl J Med. 2017 Oct 12; 377: 1438-1447. Fallah MP, Skrip LA, Dahn BT, Nyenswah TG, Flumo H, Glayweon M, Lorseh TL, Kaler SG, Higgs ES, Galvani AP. Pregnancy outcomes in Liberian women who conceived after recovery from Ebola virus disease. Lancet Glob Health. 2016 Oct; 4: e678-9. Kaler SG. Microbial peptide de-coppers mitochondria: implications for Wilson disease. J Clin Invest. 2016 Jul 1; 126: 2412-4. Yi L, Kaler SG. Direct interactions of adaptor protein complexes 1 and 2 with the copper transporter ATP7A mediate its anterograde and retrograde trafficking. Hum Mol Genet. 2015 May 1; 24: 2411-25. Bandmann O, Weiss KH, Kaler SG. Wilson's disease and other neurological copper disorders. Lancet Neurol. 2015 Jan; 14: 103-13.
View More Publications
- Sharma P, Reichert M, Lu Y, Markello TC, Adams DR, Steinbach PJ, Fuqua BK, Parisi X, Kaler SG, Vulpe CD, Anderson GJ, Gahl WA, Malicdan MCV. Biallelic HEPHL1 variants impair ferroxidase activity and cause an abnormal hair phenotype. PLoS Genet. 2019 May; 15: e1008143.
- PREVAIL III Study Group., Sneller MC, Reilly C, Badio M, Bishop RJ, Eghrari AO, Moses SJ, Johnson KL, Gayedyu-Dennis D, Hensley LE, Higgs ES, Nath A, Tuznik K, Varughese J, Jensen KS, Dighero-Kemp B, Neaton JD, Lane HC, Fallah MP. A Longitudinal Study of Ebola Sequelae in Liberia. N Engl J Med. 2019 Mar 7; 380: 924-934.
- Haddad MR, Choi EY, Zerfas PM, Yi L, Martinelli D, Sullivan P, Goldstein DS, Centeno JA, Brinster LR, Ralle M, Kaler SG. Cerebrospinal Fluid-Directed rAAV9-rsATP7A Plus Subcutaneous Copper Histidinate Advance Survival and Outcomes in a Menkes Disease Mouse Model. Mol Ther Methods Clin Dev. 2018 Sep 21; 10: 165-178.
- Yi L, Kaler SG. Interaction between the AAA ATPase p97/VCP and a concealed UBX domain in the copper transporter ATP7A is associated with motor neuron degeneration. J Biol Chem. 2018 May 18; 293: 7606-7617.
- Kennedy SB, Bolay F, Kieh M, Grandits G, Badio M, Ballou R, Eckes R, Feinberg M, Follmann D, Grund B, Gupta S, Hensley L, Higgs E, Janosko K, Johnson M, Kateh F, Logue J, Marchand J, Monath T, Nason M, Nyenswah T, Roman F, Stavale E, Wolfson J, Neaton JD, Lane HC, PREVAIL I Study Group.. Phase 2 Placebo-Controlled Trial of Two Vaccines to Prevent Ebola in Liberia. N Engl J Med. 2017 Oct 12; 377: 1438-1447.
- Fallah MP, Skrip LA, Dahn BT, Nyenswah TG, Flumo H, Glayweon M, Lorseh TL, Kaler SG, Higgs ES, Galvani AP. Pregnancy outcomes in Liberian women who conceived after recovery from Ebola virus disease. Lancet Glob Health. 2016 Oct; 4: e678-9.
- Kaler SG. Microbial peptide de-coppers mitochondria: implications for Wilson disease. J Clin Invest. 2016 Jul 1; 126: 2412-4.
- Yi L, Kaler SG. Direct interactions of adaptor protein complexes 1 and 2 with the copper transporter ATP7A mediate its anterograde and retrograde trafficking. Hum Mol Genet. 2015 May 1; 24: 2411-25.
- Bandmann O, Weiss KH, Kaler SG. Wilson’s disease and other neurological copper disorders. Lancet Neurol. 2015 Jan; 14: 103-13.
Education
Date of Appointment at Nationwide Children’s Hospital: 09/27/2019
Board Certifications
Clinical Biochemical/Molecular Genetics Clinical Genetics and Genomics
Fellowship
National Institutes of Health
Date Completed: 06/30/1989
Residency
Tufts New England Medical Center
Date Completed: 06/30/1988
Residency
Loyola University Medical Center
Date Completed: 06/30/1987
Internship
Brigham and Women’s Hospital
Date Completed: 06/30/1985
Medical School
University of Rochester School of Medicine/Dentist
Date Completed: 05/19/1984
Education
Date of Appointment at Nationwide Children’s Hospital: 09/27/2019
Board Certifications
Clinical Biochemical/Molecular Genetics Clinical Genetics and Genomics
Fellowship
National Institutes of Health
Date Completed: 06/30/1989
Residency
Tufts New England Medical Center
Date Completed: 06/30/1988
Residency
Loyola University Medical Center
Date Completed: 06/30/1987
Internship
Brigham and Women’s Hospital
Date Completed: 06/30/1985
Medical School
University of Rochester School of Medicine/Dentist
Date Completed: 05/19/1984
Education
Date of Appointment at Nationwide Children’s Hospital: 09/27/2019
Board Certifications
Clinical Biochemical/Molecular Genetics Clinical Genetics and Genomics
Fellowship
National Institutes of Health
Date Completed: 06/30/1989
Residency
Tufts New England Medical Center
Date Completed: 06/30/1988
Residency
Loyola University Medical Center
Date Completed: 06/30/1987
Internship
Brigham and Women’s Hospital
Date Completed: 06/30/1985
Medical School
University of Rochester School of Medicine/Dentist
Date Completed: 05/19/1984
Date of Appointment at Nationwide Children’s Hospital: 09/27/2019
Board Certifications
Clinical Biochemical/Molecular Genetics Clinical Genetics and Genomics
Fellowship
National Institutes of Health
Date Completed: 06/30/1989
Residency
Tufts New England Medical Center
Date Completed: 06/30/1988
Residency
Loyola University Medical Center
Date Completed: 06/30/1987
Internship
Brigham and Women’s Hospital
Date Completed: 06/30/1985
Medical School
University of Rochester School of Medicine/Dentist
Date Completed: 05/19/1984
Date of Appointment at Nationwide Children’s Hospital: 09/27/2019
Board Certifications
Clinical Biochemical/Molecular Genetics Clinical Genetics and Genomics
Fellowship
National Institutes of Health
Date Completed: 06/30/1989
Residency
Tufts New England Medical Center
Date Completed: 06/30/1988
Residency
Loyola University Medical Center
Date Completed: 06/30/1987
Internship
Brigham and Women’s Hospital
Date Completed: 06/30/1985
Medical School
University of Rochester School of Medicine/Dentist
Date Completed: 05/19/1984
- Clinical Biochemical/Molecular Genetics
- Clinical Genetics and Genomics
Professional Experience
2015 - 2019 NIH, Head, Metals Biology/Molecular Medicine Affinity Group, Intramural Research Program2013 - 2019 NIH, Chair, Trans-NIH Gene Therapy Consortium. Intramural Research Program2001 - 2010 NICHD, NIH, Clinical Director, Intramural Research Program2000 - 2001 National Institutes of Health, Deputy Associate Director for Disease Prevention, Office of the Director1999 - 2000 Children’s Hospital National Medical Center, Director, Biochemical and Molecular Genetics; Department of Laboratory Medicine, Department of Pathology1999 - 2000 Children’s Hospital National Medical Center, Vice Chair, Division of Metabolism1995 - 2000 George Washington University School of Medicine, Associate Professor of Pediatrics and Pathology, Division of Medical Genetics, Department of Pediatrics
Professional Experience
2015 - 2019 NIH, Head, Metals Biology/Molecular Medicine Affinity Group, Intramural Research Program2013 - 2019 NIH, Chair, Trans-NIH Gene Therapy Consortium. Intramural Research Program2001 - 2010 NICHD, NIH, Clinical Director, Intramural Research Program2000 - 2001 National Institutes of Health, Deputy Associate Director for Disease Prevention, Office of the Director1999 - 2000 Children’s Hospital National Medical Center, Director, Biochemical and Molecular Genetics; Department of Laboratory Medicine, Department of Pathology1999 - 2000 Children’s Hospital National Medical Center, Vice Chair, Division of Metabolism1995 - 2000 George Washington University School of Medicine, Associate Professor of Pediatrics and Pathology, Division of Medical Genetics, Department of Pediatrics
Professional Experience
2015 - 2019 NIH, Head, Metals Biology/Molecular Medicine Affinity Group, Intramural Research Program2013 - 2019 NIH, Chair, Trans-NIH Gene Therapy Consortium. Intramural Research Program2001 - 2010 NICHD, NIH, Clinical Director, Intramural Research Program2000 - 2001 National Institutes of Health, Deputy Associate Director for Disease Prevention, Office of the Director1999 - 2000 Children’s Hospital National Medical Center, Director, Biochemical and Molecular Genetics; Department of Laboratory Medicine, Department of Pathology1999 - 2000 Children’s Hospital National Medical Center, Vice Chair, Division of Metabolism1995 - 2000 George Washington University School of Medicine, Associate Professor of Pediatrics and Pathology, Division of Medical Genetics, Department of Pediatrics
2015 - 2019 NIH, Head, Metals Biology/Molecular Medicine Affinity Group, Intramural Research Program2013 - 2019 NIH, Chair, Trans-NIH Gene Therapy Consortium. Intramural Research Program2001 - 2010 NICHD, NIH, Clinical Director, Intramural Research Program2000 - 2001 National Institutes of Health, Deputy Associate Director for Disease Prevention, Office of the Director1999 - 2000 Children’s Hospital National Medical Center, Director, Biochemical and Molecular Genetics; Department of Laboratory Medicine, Department of Pathology1999 - 2000 Children’s Hospital National Medical Center, Vice Chair, Division of Metabolism1995 - 2000 George Washington University School of Medicine, Associate Professor of Pediatrics and Pathology, Division of Medical Genetics, Department of Pediatrics
2015 - 2019 NIH, Head, Metals Biology/Molecular Medicine Affinity Group, Intramural Research Program
Contact Information
Pediatrics
Call us at: (614) 722-3535
Fax us at: (614) 722-3273
Center for Gene Therapy700 Children's Drive Rm WA3021Columbus, OH 43205 (map)
Contact Information
Pediatrics
Call us at: (614) 722-3535
Fax us at: (614) 722-3273
Center for Gene Therapy700 Children's Drive Rm WA3021Columbus, OH 43205 (map)
Contact Information
Pediatrics
Call us at: (614) 722-3535
Fax us at: (614) 722-3273
Center for Gene Therapy700 Children's Drive Rm WA3021Columbus, OH 43205 (map)
Pediatrics
Call us at: (614) 722-3535
Fax us at: (614) 722-3273
Center for Gene Therapy700 Children's Drive Rm WA3021Columbus, OH 43205 (map)
Call us at: (614) 722-3535
Fax us at: (614) 722-3273
Center for Gene Therapy700 Children's Drive Rm WA3021Columbus, OH 43205 (map)
Call us at: (614) 722-3535
Fax us at: (614) 722-3273
Center for Gene Therapy700 Children's Drive Rm WA3021Columbus, OH 43205 (map)
- Call us at:
- (614) 722-3535
- Fax us at:
- (614) 722-3273
- Center for Gene Therapy700 Children’s Drive Rm WA3021Columbus, OH 43205 (map)
