Jerry R. Mendell, Ph.D.

Nationwide Children’s Hospital
Center for Gene Therapy WA-3011
700 Children’s Drive
Columbus, OH 43210



Jerry R. Mendell, Ph.D.

Education & Training:
University of Texas Southwestern Medical School, Dallas, 1966 M.D.
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
Medical Neurology Branch, NINDS, NIH, Bethesda, MD, 1972 Fellow

Research Interest:
Dr. Mendell served from 2004-2017 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.

Selected Publications:

  • Mendell JR, Rodino-Klapac L, Sahenk Z, Malik V, Kaspar BK, Walker CM, Clark KR. Gene therapy for muscular dystrophy: lessons learned and path forward. Neuroscience letters. 2012;527:90-9.
  • Mendell JR, Shilling C, Leslie ND, Flanigan KM, al-Dahhak R, Gastier-Foster J, Kneile K, Dunn DM, Duval B, Aoyagi A, Hamil C, Mahmoud M, Roush K, Bird L, Rankin C, Lilly H, Street N, Chandrasekar R, Weiss RB. Evidence-based path to newborn screening for Duchenne muscular dystrophy. Annals of neurology. 2012;71:304-13.
  • Mendell JR, Lloyd-Puryear M. Report of MDA muscle disease symposium on newborn screening for Duchenne muscular dystrophy. Muscle & nerve. 2013;48:21-6.
  • Mendell JR, Rodino-Klapac LR, Sahenk Z, Roush K, Bird L, Lowes LP, Alfano L, Gomez AM, Lewis S, Kota J, Malik V, Shontz K, Walker CM, Flanigan KM, Corridore M, Kean JR, Allen HD, Shilling C, Melia KR, Sazani P, Saoud JB, Kaye EM. Eteplirsen for the treatment of Duchenne muscular dystrophy. Annals of neurology. 2013;74:637-47.
  • Chicoine LG, Rodino-Klapac LR, Shao G, Xu R, Bremer WG, Camboni M, Golden B, Montgomery CL, Shontz K, Heller KN, Griffin DA, Lewis S, Coley BD, Walker CM, Clark KR, Sahenk Z, Mendell JR, Martin PT. Vascular delivery of rAAVrh74.MCK.GALGT2 to the gastrocnemius muscle of the rhesus macaque stimulates the expression of dystrophin and laminin alpha2 surrogates. Molecular therapy : the journal of the American Society of Gene Therapy. 2014;22:713-24.
  • Connolly AM, Florence JM, Cradock MM, Eagle M, Flanigan KM, McDonald CM, Karachunski PI, Darras BT, Bushby K, Malkus EC, Golumbek PT, Zaidman CM, Miller JP, Mendell JR. One year outcome of boys with Duchenne muscular dystrophy using the Bayley-III scales of infant and toddler development. Pediatric neurology. 2014;50:557-63.
  • Al-Zaidy SA, Sahenk Z, Rodino-Klapac LR, Kaspar B, Mendell JR. Follistatin Gene Therapy Improves Ambulation in Becker Muscular Dystrophy. Journal of neuromuscular diseases. 2015;2:185-92.
  • Connolly AM, Malkus EC, Mendell JR, Flanigan KM, Miller JP, Schierbecker JR, Siener CA, Golumbek PT, Zaidman CM, McDonald CM, Johnson L, Nicorici A, Karachunski PI, Day JW, Kelecic JM, Lowes LP, Alfano LN, Darras BT, Kang PB, Quigley J, Pasternak AE, Florence JM. Outcome reliability in non-ambulatory boys/men with Duchenne muscular dystrophy. Muscle & nerve. 2015;51:522-32.
  • Heller KN, Montgomery CL, Shontz KM, Clark KR, Mendell JR, Rodino-Klapac LR. Human alpha7 Integrin Gene (ITGA7) Delivered by Adeno-Associated Virus Extends Survival of Severely Affected Dystrophin/Utrophin-Deficient Mice. Human gene therapy. 2015;26:647-56.
  • Mendell JR, Sahenk Z, Malik V, Gomez AM, Flanigan KM, Lowes LP, Alfano LN, Berry K, Meadows E, Lewis S, Braun L, Shontz K, Rouhana M, Clark KR, Rosales XQ, Al-Zaidy S, Govoni A, Rodino-Klapac LR, Hogan MJ, Kaspar BK. A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy. Molecular therapy : the journal of the American Society of Gene Therapy. 2015;23:192-201.
  • Connolly AM, Florence JM, Zaidman CM, Golumbek PT, Mendell JR, Flanigan KM, Karachunski PI, Day JW, McDonald CM, Darras BT, Kang PB, Siener CA, Gadeken RK, Anand P, Schierbecker JR, Malkus EC, Lowes LP, Alfano LN, Johnson L, Nicorici A, Kelecic JM, Quigley J, Pasternak AE, Miller JP. Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy: MDA-DMD network follow-up. Muscle & nerve. 2016;54:681-9.
  • Harris E, Bladen CL, Mayhew A, James M, Bettinson K, Moore U, Smith FE, Rufibach L, Cnaan A, Bharucha-Goebel DX, Blamire AM, Bravver E, Carlier PG, Day JW, Diaz-Manera J, Eagle M, Grieben U, Harms M, Jones KJ, Lochmuller H, Mendell JR, Mori-Yoshimura M, Paradas C, Pegoraro E, Pestronk A, Salort-Campana E, Schreiber-Katz O, Semplicini C, Spuler S, Stojkovic T, Straub V, Takeda S, Rocha CT, Walter MC, Bushby K. The Clinical Outcome Study for dysferlinopathy: An international multicenter study. Neurology Genetics. 2016;2:e89.
  • Mendell JR, Goemans N, Lowes LP, Alfano LN, Berry K, Shao J, Kaye EM, Mercuri E. Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy. Annals of neurology. 2016;79:257-71.
  • Mendell JR, Rodino-Klapac LR. Duchenne muscular dystrophy: CRISPR/Cas9 treatment. Cell research. 2016;26:513-4.
  • Pozsgai ER, Griffin DA, Heller KN, Mendell JR, Rodino-Klapac LR. beta-Sarcoglycan gene transfer decreases fibrosis and restores force in LGMD2E mice. Gene therapy. 2016;23:57-66.
  • Alfano LN, Miller NF, Berry KM, Yin H, Rolf KE, Flanigan KM, Mendell JR, Lowes LP. The 100-meter timed test: Normative data in healthy males and comparative pilot outcome data for use in Duchenne muscular dystrophy clinical trials. Neuromuscular disorders : NMD. 2017;27:452-7.
  • Heller KN, Mendell JT, Mendell JR, Rodino-Klapac LR. MicroRNA-29 overexpression by adeno-associated virus suppresses fibrosis and restores muscle function in combination with micro-dystrophin. JCI insight. 2017;2.
  • Mendell JR, Al-Zaidy S, Shell R, Arnold WD, Rodino-Klapac LR, Prior TW, Lowes L, Alfano L, Berry K, Church K, Kissel JT, Nagendran S, L’Italien J, Sproule DM, Wells C, Cardenas JA, Heitzer MD, Kaspar A, Corcoran S, Braun L, Likhite S, Miranda C, Meyer K, Foust KD, Burghes AHM, Kaspar BK. Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy. The New England journal of medicine. 2017;377:1713-22.
  • Mendell JR, Sahenk Z, Al-Zaidy S, Rodino-Klapac LR, Lowes LP, Alfano LN, Berry K, Miller N, Yalvac M, Dvorchik I, Moore-Clingenpeel M, Flanigan KM, Church K, Shontz K, Curry C, Lewis S, McColly M, Hogan MJ, Kaspar BK. Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes. Molecular therapy : the journal of the American Society of Gene Therapy. 2017;25:870-9.
  • Potter RA, Griffin DA, Sondergaard PC, Johnson RW, Pozsgai ER, Heller KN, Peterson EL, Lehtimaki KK, Windish HP, Mittal PJ, Albrecht DE, Mendell JR, Rodino-Klapac LR. Systemic Delivery of Dysferlin Overlap Vectors Provides Long-Term Gene Expression and Functional Improvement for Dysferlinopathy. Human gene therapy. 2017.
  • 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.
  • Mendell JR. Therapy for Spinal Muscular Atrophy. The New England journal of medicine. 2018;378:487.