Aminoglycoside-induced mutation suppression (stop codon readthrough) as a therapeutic strategy for Duchenne muscular dystrophy

by Vinod Malik, Louise R. Rodino-Klapac, Laurence Viollet, Jerry R. Mendell
Published 2010-11-01

A Roadmap to Newborn Screening for Duchenne Muscular Dystrophy

by Samiah A. Al-Zaidy, Michele Lloyd-Puryear, Annie Kennedy, Veronica Lopez, Jerry R. Mendell
Published 2017-04-01

Systemic delivery of AAVrh74.tMCK.hCAPN3 rescues the phenotype in a mouse model for LGMD2A/R1

by Zarife Sahenk, Burcak Ozes, Darren Murrey, Morgan Myers, Kyle Moss, Mehmet E. Yalvac, Alicia Ridgley, Lei Chen, Jerry R. Mendell
Published 2021-09-01

Adeno-associated virus serotype 9 antibodies in patients screened for treatment with onasemnogene abeparvovec

by John W. Day, Richard S. Finkel, Eugenio Mercuri, Kathryn J. Swoboda, Melissa Menier, Rudolf van Olden, Sitra Tauscher-Wisniewski, Jerry R. Mendell
Published 2021-06-01

RNAi-mediated Gene Silencing of Mutant Myotilin Improves Myopathy in LGMD1A Mice

by Jian Liu, Lindsay M Wallace, Sara E Garwick-Coppens, Darcée D Sloboda, Carol S Davis, Chady H Hakim, Michael A Hauser, Susan V Brooks, Jerry R Mendell, Scott Q Harper
Published 2014-01-01

Homologous recombination mediates functional recovery of dysferlin deficiency following AAV5 gene transfer.

by William E Grose, K Reed Clark, Danielle Griffin, Vinod Malik, Kimberly M Shontz, Chrystal L Montgomery, Sarah Lewis, Robert H Brown, Paul M L Janssen, Jerry R Mendell, Louise R Rodino-Klapac
Published 2012-01-01

Reducing sarcolipin expression mitigates Duchenne muscular dystrophy and associated cardiomyopathy in mice

by Antanina Voit, Vishwendra Patel, Ronald Pachon, Vikas Shah, Mohammad Bakhutma, Erik Kohlbrenner, Joseph J. McArdle, Louis J. Dell’Italia, Jerry R. Mendell, Lai-Hua Xie, Roger J. Hajjar, Dongsheng Duan, Diego Fraidenraich, Gopal J. Babu
Published 2017-10-01

Intensive Teenage Activity Is Associated With Greater Muscle Hyperintensity on T1W Magnetic Resonance Imaging in Adults With Dysferlinopathy

by Ursula Moore, Marni Jacobs, Marni Jacobs, Roberto Fernandez-Torron, Roberto Fernandez-Torron, Jaume LLauger Rossello, Fiona E. Smith, Meredith James, Anna Mayhew, Laura Rufibach, Pierre G. Carlier, Andrew M. Blamire, John W. Day, Kristi J. Jones, Diana X. Bharucha-Goebel, Diana X. Bharucha-Goebel, Emmanuelle Salort-Campana, Alan Pestronk, Maggie C. Walter, Carmen Paradas, Carmen Paradas, Tanya Stojkovic, Madoka Mori-Yoshimura, Elena Bravver, Elena Pegoraro, Jerry R. Mendell, Kate Bushby, Volker Straub, Jordi Diaz-Manera, Jordi Diaz-Manera, Jordi Diaz-Manera
Published 2020-12-01