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Enhancing interaction of actin and actin-binding domain 1 of dystrophin with modulators: Toward improved gene therapy for Duchenne muscular dystrophy

Duchenne muscular dystrophy is a lethal muscle disease, caused by mutations in the gene encoding dystrophin, an actin-binding cytoskeletal protein. Absence of functional dystrophin results in muscle weakness and degeneration, eventually leading to cardiac and respiratory failure. Strategies to repla...

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Detalles Bibliográficos
Autores principales: Guhathakurta, Piyali, Carter, Anna L., Thompson, Andrew R., Kurila, Dillon, LaFrence, Jeffrey, Zhang, Li, Trask, Jake R., Vanderheyden, Bri, Muretta, Joseph M., Ervasti, James M., Thomas, David D.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society for Biochemistry and Molecular Biology 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9731851/
https://www.ncbi.nlm.nih.gov/pubmed/36372234
http://dx.doi.org/10.1016/j.jbc.2022.102675