Optimized lentiviral vector to restore full-length dystrophin via a cell-mediated approach in a mouse model of Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is a muscle wasting disorder caused by mutations in the DMD gene. Restoration of full-length dystrophin protein in skeletal muscle would have therapeutic benefit, but lentivirally mediated delivery of such a large gene in vivo has been hindered by lack of tissue spe...

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Detalles Bibliográficos
Autores principales: Meng, Jinhong, Moore, Marc, Counsell, John, Muntoni, Francesco, Popplewell, Linda, Morgan, Jennifer
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9121076/
https://www.ncbi.nlm.nih.gov/pubmed/35615709
http://dx.doi.org/10.1016/j.omtm.2022.04.015

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