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

Muscular Dystrophy Parent/ Patient Conference

Please join us for the 3rd annual Wellstone Center Muscular Dystrophy Parent/ Patient Conference!

Date: April 12, 2014
Time: 8 am to 5 pm
Location: Nationwide Children's Hospital

Learn more about the conference and register to attend »

Jerry R. Mendell, MD

Jerry R. Mendell, MD Best Doctors 2011-2012

Contact Information

Neurology
700 Children's Dr
Columbus, OH 43205 [ map ]
PH: (614) 722-5615
FX: (614) 722-3273
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

Biography

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 1990’s 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

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 dystrophy can also be treated with follistatin by way of IM injection with adeno-associated virus (AAV) vectors to carry the follistatin 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 partially functional dystrophin protein. In addition to developing potential treatments for neuromuscular diseases, the Mendell laboratory also concentrates on the diagnosis and characterization of neuromusclar diseases. The Mendell lab also serves as the diagnostic lab of muscle biopsies for the Anatomic Pathology Department 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 Nationwide Children's Hospital 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

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

Specialty:

  • Neurology

Date of Appointment at Nationwide Children’s Hospital:

  • 08/27/2004

Professional Experience

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

  • Rodino-Klapac,Louise,R; Mendell,Jerry,R; Sahenk,Zarife. 2013. Update on the treatment of Duchenne muscular dystrophy.  Current neurology and neuroscience reports. Vol. 13, no. 3. (March): 332-?.
  • Heller,Kristin,N; Montgomery,Chrystal,L; Janssen,Paul,ML; Clark,K,Reed; Mendell,Jerry,R; Rodino-Klapac,Louise,R. 2013. AAV-mediated Overexpression of Human a7 Integrin Leads to Histological and Functional Improvement in Dystrophic Mice.  Molecular therapy. Vol. 21, no. 3. (March): 520-525.
  • Flanigan KF, Ceco E, Lamar K-M, Kaminoh Y, Dunn DM, Mendell JR, King WM, Pestronk A, Florence JM, Mathews KD, Finkel RS, Swoboda KJ, Gappmaier E, Howard MT, Day JW, McDonald C, McNally EM, Weiss RB. 2012. LTBP4 genotype predicts age of ambulatory loss in Duchenne Muscular Dystrophy.  Ann Neurol. Vol. 26, no. November: eEpub ahead of print.
  • Mendell,Jerry,R; Rodino-Klapac,Louise; Sahenk,Zarife; Malik,Vinod; Kaspar,Brian,K; Walker,Christopher,M; Clark,K,Reed. 2012. Gene therapy for muscular dystrophy: Lessons learned and path forward.  NEUROSCIENCE LETTERS. Vol. 527, no. 2. (October): 90-99.
  • Rosales XQ, Malik V, Sneh A, Chen L, Lewis S, Kota J, Gastier-Foster JM, Astbury C, Pyatt R, Reshmi S, Rodino-Klapac LR, Clark KR, Mendell JR, Sahenk Z. 2012. Impaired regeneration in LGMD 2A supported by increased Pax7 positive satellite cell content and muscle specific microRNA dysregulation.  Muscle Nerve. Vol. 7, no. September: eEpub ahead of print.
  • Hsu S-H, Wang B, Kota J, Yu J, Costinean S, Kutay H, Yu L, Bai S, Perle KL, Chivukula RR, Mao H, Wei M, Clark KR, Mendell JR, Caligiuri MA, Jacob ST, Mendell JT, Ghoshal K. 2012. Essential metabolic, anti-inflammatory and anti-tumorigenic functions for miR-122 in mouse liver.  J Clin Invest. Vol. 122, no. August: 2871-83.
  • Wallace LM, Liu J, Domire JS, Garwick-Coppens SE, Guckes SM, Mendell JR, Flanigan KM, Harper SQ. 2012. RNA Interference Inhibits DUX4-induced Muscle Toxicity In Vivo: Implications for a Targeted FSHD Therapy.  Mol Ther. Vol. 20, no. July: 1417-23.
  • Viollet,Laurence; Thrush,Philip,T; Flanigan,Kevin,M; Mendell,Jerry,R; Allen,Hugh,D. 2012. Effects of Angiotensin-Converting Enzyme Inhibitors and/or Beta Blockers on the Cardiomyopathy in Duchenne Muscular Dystrophy.  AMERICAN JOURNAL OF CARDIOLOGY. Vol. 110, no. 1. (July): 98-102.
  • Grose WE, Clark KR, Griffin D, Malik V, Shontz KM, Montgomery CL, Lewis S, Brown RH Jr, Janssen PML, Mendell JR, Rodino-Klapac LR. 2012. Homologous Recombination Mediates Functional Recovery of Dysferlin Deficiency Following AAV5 Gene Transfer.  Plos One. Vol. 7, no. June: eE Pub.
  • Malik,Vinod; Rodino-Klapac,Louise,R; Mendell,Jerry,R. 2012. Emerging drugs for Duchenne muscular dystrophy.  EXPERT OPINION ON EMERGING DRUGS. Vol. 17, no. 2. (June): 261-277.
  • Cottrell,C,E; Mendell,J; Hart-Kothari,M; ELL,D; Thrush,D,L; Astbury,C; Pastore,M; Gastier-Foster,J,M; Pyatt,R,E. 2012. Maternal uniparental disomy of chromosome 4 in a patient with limb-girdle muscular dystrophy 2E confirmed by SNP array technology.  CLINICAL GENETICS. Vol. 81, no. 6. (June): 578-583.
  • Mendell,Jerry,R; Shilling,Chris; Leslie,Nancy,D; Flanigan,Kevin,M; Al-Dahhak,Roula; Gastier-Foster,Julie; Kneile,Kelley; Dunn,Diane,M; Duval,Brett; Aoyagi,Alexander; Hamil,Cindy; Mahmoud,Maha; Roush,Kandice; Bird,Lauren; Rankin,Chelsea; Lilly,Heather; Street,Natalie; Chandrasekar,Ram; Weiss,Robert,B. 2012. Evidence-Based Path to Newborn Screening for Duchenne Muscular Dystrophy.  ANNALS OF NEUROLOGY. Vol. 71, no. 3. (March): 304-313.
  • Sahenk Z; Mendell JR. 2011. The muscular dystrophies: distinct pathogenic mechanisms invite novel therapeutic approaches.  Current Rheumatology Reports. Vol. 13, no. 3. (June): e199.
  • Scharner J; Brown CA; Bower M; Iannaccone ST; Khatri IA; Escolar D; Gordon E; Felice K; Crowe CA; Grosmann C; Meriggioli MN; Asamoah A; Gordon O; Gnocchi VF; Ellis JA; Mendell JR; Zammit PS. 2011. Novel LMNA mutations in patients with Emery-Dreifuss muscular dystrophy and functional characterization of four LMNA mutations.  Human Mutation. Vol. 32, no. 2. (February): e152.
  • Peterson JM, Kline W, Canan BD, Ricca DJ, Kaspar B, Delfin DA, DiRienzo K, Clemens PR, Robbins PD, Baldwin AS, Flood P, Kaumaya P, Frietas M, Kornegay JN, Mendell JR, Rafael-Fortney JA, Guttridge DC, Janssen PML. 2011. Peptide-based inhibition of NF-kB rescues diaphragm muscle contractile dysfunction in a murine model of Duchenne muscular dystrophy.  Molec Med.
  • Rodino-Klapac LR; Montgomery CL; Mendell JR; Chicoine LG. 2011. AAV-mediated gene therapy to the isolated limb in rhesus macaques.  Methods In Molecular Biology (Clifton, N.J.). Vol. 709, no. January: e287.
  • Brown CA, Scharner J, Felice K, Meriggioli MN, Tarnopolsky M, Bower M, Zammit PS, Mendell JR, Ellis JA. 2011. Novel and recurrent EMD Mutations in Patients with Emery-Dreifuss muscular dystrophy, identify exon 2 as a mutation hot spot.  J Hum genet.
  • Mendell JR; Rodino-Klapac LR; Rosales XQ; Coley BD; Galloway G; Lewis S; Malik V; Shilling C; Byrne BJ; Conlon T; Campbell KJ; Bremer WG; Taylor LE; Flanigan KM; Gastier-Foster JM; Astbury C; Kota J; Sahenk Z; Walker CM; Clark KR. 2010. Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D.  Annals Of Neurology. Vol. 68, no. 5. (November): e629.
  • Malik V; Rodino-Klapac LR; Viollet L; Mendell JR. 2010. Aminoglycoside-induced mutation suppression (stop codon readthrough) as a therapeutic strategy for Duchenne muscular dystrophy.  Therapeutic Advances In Neurological Disorders. Vol. 3, no. 6. (November): e379.
  • Mendell JR; Campbell K; Rodino-Klapac L; Sahenk Z; Shilling C; Lewis S; Bowles D; Gray S; Li C; Galloway G; Malik V; Coley B; Clark KR; Li J; Xiao X; Samulski J; McPhee SW; Samulski RJ; Walker CM. 2010. Dystrophin immunity in Duchenne's muscular dystrophy.  The New England Journal Of Medicine. Vol. 363, no. 15. (October): e1429.
  • Mendell,Jerry,R; Campbell,Katherine; Rodino-Klapac,Louise; Sahenk,Zarife; Shilling,Chris; Lewis,Sarah; Bowles,Dawn; Gray,Steven; Li,Chengwen; Galloway,Gloria; Malik,Vinod; Coley,Brian; Clark,K,Reed; Li,Juan; Xiao,Xiao; Samulski,Jade; McPhee,Scott,W; Samulski,R,Jude; Walker,Christopher,M. 2010. Dystrophin immunity in Duchenne's muscular dystrophy.  New England journal of medicine. Vol. 363, no. 15. (October): 1429-1437.
  • Cottrell,Catherine,E; Prior,Thomas,W; Pyatt,Robert; Astbury,Caroline; Reshmi,Shalini; Bartholomew,Dennis; Atkin,Joan; Manickam,Kandamurugu; Thrush,Devon,Lamb; Pastore,Matthew; Mendell,Jerry; Tsao,Chang-Yong; Al-Dahhak,Roula; Newmeyer,Amy; Gastier-Foster,Julie,M. 2010. Unexpected Detection of Dystrophin Gene Deletions by Array Comparative Genomic Hybridization.  AMERICAN JOURNAL OF MEDICAL GENETICS PART A. Vol. 152A, no. 9. (September): 2301-2307.
  • Mendell JR; Rodino-Klapac LR; Malik V. 2010. Molecular therapeutic strategies targeting Duchenne muscular dystrophy.  Journal Of Child Neurology. Vol. 25, no. 9. (September): e1145.
  • Soltanzadeh P; Friez MJ; Dunn D; von Niederhausern A; Gurvich OL; Swoboda KJ; Sampson JB; Pestronk A; Connolly AM; Florence JM; Finkel RS; Bönnemann CG; Medne L; Mendell JR; Mathews KD; Wong BL; Sussman MD; Zonana J; Kovak K; Gospe SM Jr; Gappmaier E; Taylor LE; Howard MT; Weiss RB; Flanigan KM. 2010. Clinical and genetic characterization of manifesting carriers of DMD mutations.  Neuromuscular Disorders: NMD. Vol. 20, no. 8. (August): e499.
  • Rosales XQ; Gastier-Foster JM; Lewis S; Vinod M; Thrush DL; Astbury C; Pyatt R; Reshmi S; Sahenk Z; Mendell JR. 2010. Novel diagnostic features of dysferlinopathies.  Muscle & Nerve. Vol. 42, no. 1. (July): e14.
  • 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. 2010. Gentamicin-induced readthrough of stop codons in Duchenne muscular dystrophy.  Annals Of Neurology. Vol. 67, no. 6. (June): e771.
  • Saechao C; Valles-Ayoub Y; Esfandiarifard S; Haghighatgoo A; No D; Shook S; Mendell JR; Rosales-Quintero X; Felice KJ; Morel CF; Pietruska M; Darvish D. 2010. Novel GNE mutations in hereditary inclusion body myopathy patients of non-Middle Eastern descent.  Genetic Testing And Molecular Biomarkers. Vol. 14, no. 2. (April): e157.
  • Rodino-Klapac LR; Montgomery CL; Bremer WG; Shontz KM; Malik V; Davis N; Sprinkle S; Campbell KJ; Sahenk Z; Clark KR; Walker CM; Mendell JR; Chicoine LG. 2010. Persistent expression of FLAG-tagged micro dystrophin in nonhuman primates following intramuscular and vascular delivery.  Molecular Therapy: The Journal Of The American Society Of Gene Therapy. Vol. 18, no. 1. (January): e109.
  • Rosales XQ, Gastier-Foster JM, Lewis S, Malik V, Thrush DL, Astbury C, Pyatt R, Reshmi S, Sahenk Z, Mendell JR. Novel Diagnostic Features of Dysferlinopathies.  Muscle Nerve.
  • Mendell JR, Rodino-Klapac LR, Malik V. 2010. Molecular Therapeutic Strategies Targeting Duchenne Muscular Dystrophy.  J Child Neurol.
  • Mendell JR, Campbell K, rodino-Klapac L, Sahenk Z, Shilling C, Lewis S, Bwles D, Gray S, Chengwen L, Galloway G, Malik V, Coley B, Clark R, Li J, Xiao X, Samulski J, McPhee SW, Samulski RJ, Walker CM. 2010. Dystrophin Immunity Revealed by Gene therapy in Duchenne Muscular Dystrophy.  N Engl J Med.
  • Kaspar RW; Allen HD; Ray WC; Alvarez CE; Kissel JT; Pestronk A; Weiss RB; Flanigan KM; Mendell JR; Montanaro F. 2009. Analysis of dystrophin deletion mutations predicts age of cardiomyopathy onset in becker muscular dystrophy.  Circulation. Cardiovascular Genetics. Vol. 2, no. 6. (December): e544.
  • Flanigan KM; Dunn DM; von Niederhausern A; Soltanzadeh P; Gappmaier E; Howard MT; Sampson JB; Mendell JR; Wall C; King WM; Pestronk A; Florence JM; Connolly AM; Mathews KD; Stephan CM; Laubenthal KS; Wong BL; Morehart PJ; Meyer A; Finkel RS; Bonnemann CG; Medne L; Day JW; Dalton JC; Margolis MK; Hinton VJ; Weiss RB. 2009. Mutational spectrum of DMD mutations in dystrophinopathy patients: application of modern diagnostic techniques to a large cohort.  Human Mutation. Vol. 30, no. 12. (December): e1657.
  • 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. 2009. Follistatin gene delivery enhances muscle growth and strength in nonhuman primates.  Science Translational Medicine. Vol. 1, no. 6. (November): e6ra15.
  • Elsheikh B; Prior T; Zhang X; Miller R; Kolb SJ; Moore D; Bradley W; Barohn R; Bryan W; Gelinas D; Iannaccone S; Leshner R; Mendell JR; Mendoza M; Russman B; Smith S; King W; Kissel JT. 2009. An analysis of disease severity based on SMN2 copy number in adults with spinal muscular atrophy.  Muscle & Nerve. Vol. 40, no. 4. (October): e652.
  • Viollet L; Gailey S; Thornton DJ; Friedman NR; Flanigan KM; Mahan JD; Mendell JR. 2009. Utility of cystatin C to monitor renal function in Duchenne muscular dystrophy.  Muscle & Nerve. Vol. 40, no. 3. (September): e438.
  • 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. 2009. Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins.  Annals Of Neurology. Vol. 66, no. 3. (September): e290.
  • Elsheikh B; Kissel JT; Christoforidis G; Wicklund M; Kehagias DT; Chiocca EA; Mendell JR. 2009. Spinal angiography and epidural venography in juvenile muscular atrophy of the distal arm "Hirayama disease".  Muscle & Nerve. Vol. 40, no. 2. (August): e206.
  • 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. 2009. Therapeutic microRNA delivery suppresses tumorigenesis in a murine liver cancer model.  Cell. Vol. 137, no. 6. (June): e1005.
  • Rodino-Klapac LR; Haidet AM; Kota J; Handy C; Kaspar BK; Mendell JR. 2009. Inhibition of myostatin with emphasis on follistatin as a therapy for muscle disease.  Muscle & Nerve. Vol. 39, no. 3. (March): e283.
  • Martin PT; Xu R; Rodino-Klapac LR; Oglesbay E; Camboni M; Montgomery CL; Shontz K; Chicoine LG; Clark KR; Sahenk Z; Mendell JR; Janssen PM. 2009. Overexpression of Galgt2 in skeletal muscle prevents injury resulting from eccentric contractions in both mdx and wild-type mice.  American Journal Of Physiology. Cell Physiology. Vol. 296, no. 3. (March): eC476.
  • Tsao CY; Mendell JR. 2009. Partial epilepsy in an adolescent male with limb-girdle muscular dystrophy 1B.  Journal Of Child Neurology. Vol. 24, no. 3. (March): e346.
  • Flanigan, KM, Dunn DM, von Niederman A, Howard MT, Mendell JR, Connolly A, Saunders C, Modricin A,. DMD Trp 3X nonsense mutaton associated with a founder effect in North American families with mild Becker Muscular Dystrophy.  Neuromuscul Disord.
  • Guenther UP; Handoko L; Varon R; Stephani U; Tsao CY; Mendell JR; Lützkendorf S; Hübner C; von Au K; Jablonka S; Dittmar G; Heinemann U; Schuetz A; Schuelke M. 2009. Clinical variability in distal spinal muscular atrophy type 1 (DSMA1): determination of steady-state IGHMBP2 protein levels in five patients with infantile and juvenile disease.  Journal Of Molecular Medicine (Berlin, Germany). Vol. 87, no. 1. (January): e31.
  • Thrush PT; Allen HD; Viollet L; Mendell JR. 2009. Re-examination of the electrocardiogram in boys with Duchenne muscular dystrophy and correlation with its dilated cardiomyopathy.  The American Journal Of Cardiology. Vol. 103, no. 2. (January): e262.
  • Rodino-Klapac LR; Lee JS; Mulligan RC; Clark KR; Mendell JR. 2008. Lack of toxicity of alpha-sarcoglycan overexpression supports clinical gene transfer trial in LGMD2D.  Neurology. Vol. 71, no. 4. (July): e240.
  • Rosales XQ; Chu ML; Shilling C; Wall C; Pastores GM; Mendell JR. 2008. Fidelity of gamma-glutamyl transferase (GGT) in differentiating skeletal muscle from liver damage.  Journal Of Child Neurology. Vol. 23, no. 7. (July): e748.
  • 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; Allen R; Parsons SA; Wozney JM; Lavallie ER; Mendell JR. 2008. A phase I/IItrial of MYO-029 in adult subjects with muscular dystrophy.  Annals Of Neurology. Vol. 63, no. 5. (May): e561.
  • Haidet AM; Rizo L; Handy C; Umapathi P; Eagle A; Shilling C; Boue D; Martin PT; Sahenk Z; Mendell JR; Kaspar BK. 2008. Long-term enhancement of skeletal muscle mass and strength by single gene administration of myostatin inhibitors.  Proceedings Of The National Academy Of Sciences Of The United States Of America. Vol. 105, no. 11. (March): e4318.
  • Kannanayakal TJ; Mendell JR; Kuret J. 2008. Casein kinase 1 alpha associates with the tau-bearing lesions of inclusion body myositis.  Neuroscience Letters. Vol. 431, no. 2. (January): e141.
  • Tsao CY; Mendell JR. 2007. Autistic disorder in 2 children with mitochondrial disorders.  Journal Of Child Neurology. Vol. 22, no. 9. (September): e1121.
  • Rodino-Klapac LR; Chicoine LG; Kaspar BK; Mendell JR. 2007. Gene therapy for duchenne muscular dystrophy: expectations and challenges.  Archives Of Neurology. Vol. 64, no. 9. (September): e1236.
  • King WM; Ruttencutter R; Nagaraja HN; Matkovic V; Landoll J; Hoyle C; Mendell JR; Kissel JT. 2007. Orthopedic outcomes of long-term daily corticosteroid treatment in Duchenne muscular dystrophy.  Neurology. Vol. 68, no. 19. (May): e1607.
  • Mendell JR; Csimma C; McDonald CM; Escolar DM; Janis S; Porter JD; Hesterlee SE; Howell RR. 2007. Challenges in drug development for muscle disease: a stakeholders' meeting.  Muscle & Nerve. Vol. 35, no. 1. (January): e8.
  • Mendell,Jerry,R; Sahenk,Zarife. 2003. Clinical practice. Painful sensory neuropathy.  New England journal of medicine. Vol. 348, no. 13. (March): 1243-1255.
  • Mendell JR, Lloyd-Puryear M. Report of MDA Muscle Disease Symposium on Newborn Screening for Duchenne Muscular Dystrophy.  Muscle and Nerve.
  • Boyden SE, Mahoney L, Kawahara G, Myers JA, Estrella EA, Mitsuhashi S, Duncan AR, Dey F, deChene T, Blasko-Goehringer JM, Darras BT, Mendell JR, Lidov HGW, Nishino I, Beggs AH, Kunkel LM, Kang PB. Mutations in the satellite cell gene MEGF10 cause a recessive congenital myopathy with minicores.  Neurogenetics.
  • Allen HD, Thrush PT, Hoffman TM, Flanigan KM, Mendell JR. Cardiac Management in Neuromuscular Diseases.  Phys Med Rehabil Clin N Am.
  • Connolly AM, Florence JM, Cradock MM, Malkus EC, Schierbecker JR, Siene CA, Wulf CO, Anand P, Golumbek PT, Zaidman CM, Miller JP, Lowes P, Alfano LN, Viollet-Callendret L, Flanigan KM, Mendell JR, McDonald CM, Goude E, Johnson L, Nicorici A,. Motor and Cognitive Assessment of Infants and Young Boys with Duchenne Muscular Dystrophy;Results from the Muscular Dystrophy Association DMD Clinical Research Network.  Muscle and Nerve.
  • Mendell JR, Rodino-Klapac LR, Malik V. Molecular Therapeutic Strategies Targeting Duchenne Muscular Dystrophy.  J Child Neurol.
  • Alfano LN, Lowes LP, Flanigan KM, Mendell JR. Correlation of lower extremity strength to performance outcomes in Becker muscular dystrophy.  Muscle Nerve.
  • Lowes LP, Alfano L, Viollet L, Rosales XQ, Sahenk, Z, Kaspar BK, Clark KR, Flanigan KM, Mendell JR, McDermott,MP. Knee extensor strength predicts function in sporadic inclusion body myositis.  Muscle and Nerve. PubMed ID: 23553538
  • Thrush PT, Edward N, Flanigan KM, Mendell JR, Allen HD. Precordial R wave height does not correlate with echocardiographic findings in boys with Duchenne muscular dystrophy.  Cong Heart Dis.

Awards, Honors and Organizations

  • Presented with the Juanita Curran and Dwight Peters Endowed Chair (2010)
  • Distinguished Scholar Award presented by the president of The Ohio State University (2009)
  • S. Mouchly Small Scientific Achievement Award from the Muscular Dystrophy Association for significant contributions to neuromuscular disease research (2004)
  • Listed, “Best Doctors in America”
Nationwide Children's Hospital
700 Children's Drive Columbus, Ohio 43205 614.722.2000