Jerry R. Mendell, M.D.
Nationwide Children’s Hospital
Center for Gene Therapy WA-3011
700 Children’s Drive
Columbus, OH 43210
Education & Training:
University of Texas Southwestern Medical School, Dallas, 1966
New York Neurological Institute, Columbia University College of Physicians
and Surgeons, 1967 Internship
New York Neurological Institute, Columbia
University College of Physicians and Surgeons, 1969 Resident
Branch, NINDS, NIH, Bethesda, MD, 1972 Fellow
Dr. Mendell currently serves as Director of the Center for Gene
Therapy at The Research Institute at Nationwide Childrens Hospital and
holds Professorships in Neurology, Pediatrics, Pathology, Physiology and
Cell Biology at the Ohio State University. He also directs the neuromuscular
research program at Nationwide Children’s Hospital. He has
worked in the laboratory using experimental models of muscular dystrophy
and at the bedside participating in clinical trials. He was a founding
member of the study group known as "The Clinical Investigation of
Duchenne Dystrophy (CIDD)" that defined testing methods for clinical
trials in boys with Duchenne muscular dystrophy (DMD), delineated the natural
history of the disease, and tested many pharmacologic agents including
corticosteroids, the only treatment known to improve the natural history
of the disease. Dr. Mendell is the first to perform gene therapy
for DMD (March 2007) and also started a gene therapy study in LGMD2D (Nov
2007), demonstrating success for the first time. His laboratory also
recently demonstrated vascular delivery by rAAV of muscular dystrophy genes
to the limbs of mice and non-human primates that will be moving toward
clinical trials to make a difference in the lives of patients with devastating
disease of muscle.
In 2004, Dr. Mendell received the highest honor that
MDA bestows on physicians and scientists for contributions to muscular
dystrophy research (Scientific Achievement Award). In 2005 he received
honorary lifetime membership in the American Association of Neuromuscular
Disease and Electromyography for outstanding contributions to the field
of muscular dystrophy. In
2006, he was recognized by the American Academy of Neurology for clinical
and scientific contributions to the muscular dystrophy field. In
2009, he was presented the Distinguished Scholar Award by the President
of The Ohio State University.
- Barohn, R.J., Levine, E.J., Olson, J.O., Mendell, J.R.: Gastric
Hypomotility in Duchenne's Muscular Dystrophy - A Possible Manifestation
of Smooth Muscle
Dystrophin Deficiency. N Engl J Med 1989, 319:15-18.
- Mendell, J.R., Moxley, R.T., Griggs, R.C., Brooke, M.H., Fenichel,
G.M., Miller, J.P., King, W., Signore, L., Pandya, S., Florence, J.,
Schierbecker, J., Robison, J., Kaiser, K., Mandel, S., Arfken, C.,
Gilder, B. Randomized, Double-Blind Six-Month Trial of Prednisone
in Duchenne's Muscular Dystrophy. N Engl J Med 1989,
- Stedman H, Wilson JM, Finke R, Klenke A-L, Mendell JR. Clinical
Protocol.Phase I clinical trial utilizing gene therapy for limb girdle
muscular dystrophy: a-, b-,g-, or d-sarcoglycan gene delivered with
intramuscular instillationsof adeno-associated vectors. Human
Gene Therapy 2000, 11:777-790.
- Mendell JR, Buzin CH, Feng J, Yan J, Serrano C, Sangani DS, Prior
TW, Sommer SS. Diagnosis of Duchenne Dystrophy by Enhanced Detection
of Small Mutations. Neurology 2001, 57:645-50.
- Flanigan KM, von Niederausern A, Dunn DM, Alder J, Mendell JR, Weiss
RB. Rapid Direct Sequence Analysis of the Dystrophin Gene. Am J
- Mendell JR, Miller A. Gene Transfer for Neurological Disease: Agencies,
Policies, and Process. Neurology 2004, 63:2203-2208
- Mendell JR, Clark KR. Risks, benefits, and consent in the age
of gene therapy. Neurology 2006, 6:47-56.
- Mendell JR, Boue DR, Martin PT. The congenital muscular dystrophies: recent
advances and molecular insights. Pediatric and Developmental Pathology
- Moore SA, Shilling CJ, Westra S, Wall C, Wicklund MP, Stolle C, Brown
CA, Michele DE, Piccolo F, Winder TL, Stence A, Barresi R, King N,
King W, Florence J, Campbell KP, Fenichel GM, Stedman JJ, Kissel JT,
Griggs RC, Pandya S, Mathews KD, Pestronk A, Serrano C, Darvish D,
Mendell JR. Limb girdle muscular dystrophy in the United States. J
Neuropath Exp Neurol 2006, 65:995-1003.
- Rodino-Klapac L, Chicoine L, Kaspar B, Mendell JR. Gene Therapy for
Duchenne Muscular Dystrophy: Expectations and Challenges. Arch
Neurol 2007, 64:1-6
- Rodino-Klapac L, Janssen PML, Montgomery CL, Coley BD, Chicoine LG,
Clark KR, Mendell JR. A Translational Approach for Limb Vascular Delivery
of the Micro-dystrophin Gene without High Volume or High Pressure for
Treatment of Duchenne Muscular Dystrophy. J Transl Med 2007,
- Rodino-Klapac LR, Lee J-S, Mulligan RC, Clark KR, Mendell JR. Lack
of toxicity of alpha-sarcoglycan overexpression supports clinical gene
transfer in LGMD2D. Neurology, 2008, 71:240-247
- Wagner KR, Fleckenstein JL, Amato AA, Barohn RJ, Bushby K, Escolar
DM, Flanigan KM, Pestronk A, Tawil R, Wolfe GI, Eagle M,Florence JM,
King WM, Pandya
S, Straub V, Juneau P, Meyers K, Csimma C, Araujo T, Parsons
SA, Wozney JM, LaVallie ER, Mendell JR. A Phase I/II trial
of MYO-029 in adult subjects with muscular dystrophy. Ann Neurol 2008:63:543-545.
- Haidet AM, Rizo L, Handy C, Umapathi P, Eagle A, Shilling C, Boue
D, Martin PT, Sahenk Z, Mendell JR, Kaspar BK. Long-term enhancement
of skeletal muscle mass and strength by single gene administration
of myostatin inhibitors.
Proc Nat Acad Sci USA 2008:105:4318-4322.
- Kota J, Chivukula RR, O'Donnell KA, Wentzel EA, Montgomery CL, Hwang HW, Chang TC, Vivekanandan P, Torbenson M, Clark KR, Mendell JR, Mendell JT. Therapeutic microRNA delivery suppresses tumorigenesis in a murine liver cancer model. Cell. 2009, 137:1005-1017.
- Mendell JR, Rodino-Klapac LR, Rosales-Quintero X, Kota J, Coley BD, Galloway G, Craenen JM, Lewis S, Malik V, Shilling C, Byrne BJ, Conlon T, Campbell KJ, Bremer WG, Viollet L, Walker CM, Sahenk Z, Clark KR. Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins. Ann Neurol. 2009, 66:290-297.
- Kota J, Handy CR, Haidet AM, Montgomery CL, Eagle A, Rodino-Klapac LR, Tucker D, Shilling CJ, Therlfall WR, Walker CM, Weisbrode SE, Janssen PM, Clark KR, Sahenk Z, Mendell JR, Kaspar BK. Follistatin gene delivery enhances muscle growth and strength in nonhuman primates. Sci Transl Med. 2009, 1(6):6ra15.
Malik V, Rodino-Klapac LR, Viollet L, Wall C, King W, Al-Dahhak R, Lewis S, Shilling CJ, Kota J, Serrano-Munuera C, Hayes J, Mahan JD, Campbell KJ, Banwell B, Dasouki M, Watts V, Sivakumar K, Bien-Willner R, Flanigan KM, Sahenk Z, Barohn RJ, Walker CM, Mendell JR. Gentamicin-induced readthrough of stop codons in Duchenne muscular dystrophy. Ann Neurol. 2010, 67:771-780.
Rosales XQ, Gastier-Foster JM, Lewis S, Vinod M, Thrush DL, Astbury C, Pyatt R, Reshmi S, Sahenk Z, Mendell JR. Novel diagnostic features of dysferlinopathies. Muscle Nerve. 2010, 42:14-21