Mendell, Jerry R.

Evidence-based path to newborn screening for Duchenne muscular dystrophy

Mendell, J. R., Shilling, C., Leslie, N. D., Flanigan, K. M., al‐Dahhak, R., Gastier‐Foster, J., Kneile, K., Dunn, D. M., Duval, B., Aoyagi, A., et al. (2012) · Annals of Neurology

Muscle Physiology and DisordersGenomics and Rare Diseases

Cited by 813

Dystrophin Immunity in Duchenne's Muscular Dystrophy

Mendell, J. R., Campbell, K., Rodino-Klapac, L., Sahenk, Z., Shilling, C., Lewis, S., Bowles, D., Gray, S., Li, C., Galloway, G., et al. (2010) · New England Journal of Medicine

Muscle Physiology and DisordersVirus-based gene therapy research

Cited by 655

A phase I/IItrial of MYO‐029 in adult subjects with muscular dystrophy

Wagner, K. R., Fleckenstein, J. L., Amato, A. A., Barohn, R. J., Bushby, K., Escolar, D. M., Flanigan, K. M., Pestronk, A., Tawil, R., Wolfe, G. I., et al. (2008) · Annals of Neurology

Muscle Physiology and DisordersNeurogenetic and Muscular Disorders Research

Cited by 481

Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial

Mendell, J. R., Sahenk, Z., Lehman, K., Nease, C., Lowes, L. P., Miller, N. F., Iammarino, M. A., Alfano, L. N., Nicholl, A., Al-Zaidy, S., et al. (2020) · JAMA Neurology

Virus-based gene therapy researchNeurogenetic and Muscular Disorders Research

Cited by 405

Sustained alpha‐sarcoglycan gene expression after gene transfer in limb‐girdle muscular dystrophy, type 2D

Mendell, J. R., Rodino‐Klapac, L. R., Rosales, X. Q., Coley, B. D., Galloway, G., Lewis, S., Malik, V., Shilling, C., Byrne, B. J., Conlon, T., et al. (2010) · Annals of Neurology

Virus-based gene therapy researchMuscle Physiology and Disorders

Cited by 248

Inhibition of myostatin with emphasis on follistatin as a therapy for muscle disease

Rodino‐Klapac, L. R., Haidet, A. M., Kota, J., Handy, C., Kaspar, B. K., & Mendell, J. R. (2009) · Muscle & Nerve

Muscle Physiology and DisordersNeurogenetic and Muscular Disorders Research

Cited by 194

Anti-dystrophin T cell responses in Duchenne muscular dystrophy: prevalence and a glucocorticoid treatment effect

Flanigan, K. M., Campbell, K., Viollet, L., Wang, W., Gomez, A. M., Walker, C. M., & Mendell, J. R. (2013) · Human Gene Therapy

Muscle Physiology and DisordersNeurogenetic and Muscular Disorders Research

Cited by 115

AAV.Dysferlin Overlap Vectors Restore Function in Dysferlinopathy Animal Models

Sondergaard, P. C., Griffin, D. A., Pozsgai, E. R., Johnson, R. W., Grose, W. E., Heller, K. N., Shontz, K. M., Montgomery, C. L., Liu, J., Clark, K. R., et al. (2015) · Annals of Clinical and Translational Neurology

Virus-based gene therapy researchNeurogenetic and Muscular Disorders Research

Cited by 99

Systemic AAV-Mediated β-Sarcoglycan Delivery Targeting Cardiac and Skeletal Muscle Ameliorates Histological and Functional Deficits in LGMD2E Mice

Pozsgai, E. R., Griffin, D. A., Heller, K. N., Mendell, J. R., & Rodino-Klapac, L. R. (2017) · Molecular Therapy

Virus-based gene therapy researchMuscle Physiology and Disorders

Cited by 76

Homologous Recombination Mediates Functional Recovery of Dysferlin Deficiency following AAV5 Gene Transfer

Grose, W. E., Clark, K. R., Griffin, D., Malik, V., Shontz, K. M., Montgomery, C. L., Lewis, S., Brown, R. H., Janssen, P. M. L., Mendell, J. R., et al. (2012) · PLoS ONE

Virus-based gene therapy researchNeurogenetic and Muscular Disorders Research

Cited by 76