Jerry R. Mendell, MD :: Nationwide Children’s Hospital, Columbus, Ohio

Jerry R. Mendell, MD

Jerry R. Mendell, MD Best Doctors 2011-2012

Center for Gene Therapy
Director

Neurology
Physician Team

Neuromuscular Disorders
Director

Neuromuscular Genetic Therapeutics Fellowship
Program Co-Director

Neurosciences
Director

Contact Information

Research II - Rm WA3011
700 Children's Dr
Columbus, OH 43205 [ map ]
PH: (614) 722-2203
FX: (614) 355-5247
E-mail Me
The Research Institute at Nationwide Children's Hospital
700 Childrens Drive
Columbus, OH 43205 [ map ]
PH: (614) 722.2715
FX: (614) 355.5247

Office Practice Information:

  • Accepts patients 0-18.
  • Accepts patients 18 years or older.
  • Accepts Medicaid.
  • Requires a written physical referral.
  • This office is wheelchair accessible.

Biography

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Jerry R. Mendell, MD, is an attending neurologist at Nationwide Children’s, Director of the Center for Gene Therapy at The Research Institute of Nationwide Children’s, Director of the Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, Co-director of the Muscular Dystrophy Association clinic, and Professor of Pediatrics, Neurology, Pathology, and Physiology and Cell Biology at The Ohio State University College of Medicine. Dr. Mendell was among the first to test muscle cell transplantation for Duchenne muscular dystrophy in the early 1990s and the first person to study viral mediated gene therapy for muscular dystrophy in humans. Dr. Mendell has made fundamental contributions in clinical research and in the molecular genetics of neuromuscular disease. He has published major textbooks on the disorders of muscle and peripheral nerves along with more than 300 scientific articles and book chapters. In 2004, Dr. Mendell was awarded the S. Mouchly Small Scientific Achievement Award from the Muscular Dystrophy Association in recognition of his significant contributions to neuromuscular disease research. In 2009 he was presented the Distinguished Scholar Award by the president of The Ohio State University. Dr. Mendell is named among the “Best Doctors in America.”

Gender:

  • Male

Languages Spoken:

  • English

Research Interests

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Research Center:

Areas of Interest:

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

Research Funding*:

  • Diverse Strategies to Correct the Dystrophin Gene Using Vascular Delivery, National Institute of Neurological Disorders and Stroke
  • Gentamicin Trial in Duchenne and Limb Girdle Muscular Dystrophies, National Institute of Neurological Disorders and Stroke
  • Implementing Newborn Screening for Duchenne Muscular Dystrophy to the Community, Centers for Disease Control and Prevention
  • Phase I Study of Mini-Dystrophin Gene in AAV, Anonymous/Muscular Dystrophy
    Association, Inc.
  • Early Screening and Diagnosis of Duchenne Muscular Dystrophy, Centers for Disease Control and Prevention
  • Clinical Thresholds and Gene Transfer in DMD and LGMD, University of Pittsburgh/National Institutes of Health
  • Study for Patients with Becker, Fascioscapulohumoral and Limb-Girdle Muscular Dystrophy, Anonymous
  • Study of Mini-Dystrophin Gene in AAV, Anonymous/Muscular Dystrophy
    Association Inc.
  • Vascular Approach to Gene Therapy for Muscular Dystrophy, Children’s National Medical Center/Department of Defense
  • Immunogenicity of Golden Retrievers and Normal Dogs to rAAV Vectors Carrying Mini-Dystrophin, Muscular Dystrophy Association
  • Transfer of Alpha-Sarcoglycan Gene to LGMD2D Patients, Muscular Dystrophy Association, Inc.

Education and Training

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Medical School

  • University of Texas Southwestern Medical Center
    Date Completed: 06/30/1966

Internship

  • New York Neurological Institute, Columbia University College of Physicians and Surgeons
    Date Completed: 06/30/1967

Residency

  • Columbia Presbyterian Medical Center - New York
    Date Completed: 06/30/1969

Residency

  • National Institutes of Health
    Date Completed: 06/30/1970

Fellowship

  • National Institutes of Health
    Date Completed: 07/31/1971

Department:

  • Pediatrics

Section:

  • Neurology

Speciality:

  • Neurology, Certified

Date of Appointment at Children's Hospital:

  • 08/27/2004

Professional Experience

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present

  • Dept of Pediatrics, The Ohio State University, Professor

present

  • Dept of Pathology, The Ohio State University, Professor

present

  • Dept of Neurology, The Ohio State University, Kurtz Professor

1992–2004

  • Dept of Neurology, The Ohio State University, Chair

Publications

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Nationwide Children's Hospital
700 Children's Drive Columbus, Ohio 43205 614.722.2000