Reply to Letter to the Editor

di Mendell, Jerry R.
Pubblicato in Mol Ther (2017)

Duchenne muscular dystrophy: CRISPR/Cas9 treatment

di Mendell, Jerry R, Rodino-Klapac, Louise R
Pubblicato in Cell Res (2016)

Casein Kinase 1 alpha associates with the tau-bearing lesions of inclusion body myositis

di Kannanayakal, Theresa J., Mendell, Jerry R., Kuret, Jeff
Pubblicazione 2007

The Congenital Muscular Dystrophies: Recent Advances and Molecular Insights

di Mendell, Jerry R., Boué, Daniel R., Martin, Paul T.
Pubblicazione 2006

Emerging Drugs for Duchenne Muscular Dystrophy

di Malik, Vinod, Rodino-Klapac, Louise, Mendell, Jerry R.
Pubblicazione 2012

Could gene therapy be the future for muscular dystrophy?

di Haidet, Amanda M, Mendell, Jerry R, Kaspar, Brian K
Pubblicazione 2010

Molecular Therapeutic Strategies Targeting Duchenne Muscular Dystrophy

di Mendell, Jerry R., Rodino-Klapac, Louise R., Malik, Vinod
Pubblicazione 2010

Gene Therapy for Muscular Dystrophy: Moving the Field Forward

di Al-Zaidy, Samiah, Rodino-Klapac, Louise, Mendell, Jerry R
Pubblicato in Pediatr Neurol (2014)

Update on the Treatment of Duchenne Muscular Dystrophy

di Rodino-Klapac, Louise R., Mendell, Jerry R., Sahenk, Zarife
Pubblicato in Curr Neurol Neurosci Rep (2013)

Evaluatíon and treatment of myopathies /

di Griggs, Robert C., Mendell, Jerry R., Miller, Robert G.
Pubblicazione 1995.

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

di Malik, Vinod, Rodino-Klapac, Louise R., Viollet, Laurence, Mendell, Jerry R.
Pubblicazione 2010

MicroRNA-29 overexpression by adeno-associated virus suppresses fibrosis and restores muscle function in combination with micro-dystrophin

di Heller, Kristin N., Mendell, Joshua T., Mendell, Jerry R., Rodino-Klapac, Louise R.
Pubblicato in JCI Insight (2017)

A Roadmap to Newborn Screening for Duchenne Muscular Dystrophy

di Al-Zaidy, Samiah A., Lloyd-Puryear, Michele, Kennedy, Annie, Lopez, Veronica, Mendell, Jerry R.
Pubblicato in Int J Neonatal Screen (2017)

Follistatin Gene Therapy Improves Ambulation in Becker Muscular Dystrophy

di Al-Zaidy, Samiah A., Sahenk, Zarife, Rodino-Klapac, Louise R., Kaspar, Brian, Mendell, Jerry R.
Pubblicato in J Neuromuscul Dis (2015)

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

di Pozsgai, Eric R., Griffin, Danielle A., Heller, Kristin N., Mendell, Jerry R., Rodino-Klapac, Louise R.
Pubblicato in Mol Ther (2017)

Rapid Direct Sequence Analysis of the Dystrophin Gene

di Flanigan, Kevin M., Niederhausern, Andrew von, Dunn, Diane M., Alder, Jonathan, Mendell, Jerry R., Weiss, Robert B.
Pubblicazione 2003

INHIBITION OF MYOSTATIN WITH EMPHASIS ON FOLLISTATIN AS A THERAPY FOR MUSCLE DISEASE

di RODINO-KLAPAC, LOUISE R., HAIDET, AMANDA M., KOTA, JANAIAH, HANDY, CHALONDA, KASPAR, BRIAN K., MENDELL, JERRY R.
Pubblicazione 2009

Long-Term Pulmonary Function in Duchenne Muscular Dystrophy: Comparison of Eteplirsen-Treated Patients to Natural History

di Kinane, T. Bernard, Mayer, Oscar H., Duda, Petra W., Lowes, Linda P., Moody, Stephanie L., Mendell, Jerry R.
Pubblicato in J Neuromuscul Dis (2018)

Anti-Dystrophin T Cell Responses in Duchenne Muscular Dystrophy: Prevalence and a Glucocorticoid Treatment Effect

di Flanigan, Kevin M., Campbell, Katie, Viollet, Laurence, Wang, Wei, Gomez, Ana Maria, Walker, Christopher M., Mendell, Jerry R.
Pubblicazione 2013

Utility of Cystatin C to monitor renal function in Duchenne muscular dystrophy

di Viollet, Laurence, Gailey, Susan, Thornton, David J., Friedman, Neil R., Flanigan, Kevin M., Mahan, John D., Mendell, Jerry R.
Pubblicazione 2009