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

• Published in: Human gene therapy Vol. 24; no. 9; p. 797
• Main Authors: Flanigan, Kevin M, Campbell, Katie, Viollet, Laurence, Wang, Wei, Gomez, Ana Maria, Walker, Christopher M, Mendell, Jerry R
• Format: Journal Article
• Language: English
• Published: United States 01.09.2013
• Subjects: Age Factors; CD4-Positive T-Lymphocytes - immunology; CD8-Positive T-Lymphocytes - immunology; Dependovirus - genetics; Dystrophin - genetics; Dystrophin - immunology; Genetic Therapy; Glucocorticoids - therapeutic use; Humans; Immunosuppressive Agents - therapeutic use; Muscular Dystrophy, Duchenne - drug therapy; Muscular Dystrophy, Duchenne - immunology; Prednisone - therapeutic use; Pregnenediones - therapeutic use

Duchenne muscular dystrophy (DMD) typically occurs as a result of truncating mutations in the DMD gene that result in a lack of expression of the dystrophin protein in muscle fibers. Various therapies under development are directed toward restoring dystrophin expression at the subsarcolemmal membran...