Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is an X-linked disease caused by loss-of-function mutations in the dystrophin gene and is characterized by muscle wasting and early mortality. Adeno-associated virus-mediated gene therapy is being investigated as a treatment for DMD. In the nonclinical study documen...

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Detalles Bibliográficos
Autores principales: Le Guiner, Caroline, Xiao, Xiao, Larcher, Thibaut, Lafoux, Aude, Huchet, Corinne, Toumaniantz, Gilles, Adjali, Oumeya, Anegon, Ignacio, Remy, Séverine, Grieger, Josh, Li, Juan, Farrokhi, Vahid, Neubert, Hendrik, Owens, Jane, McIntyre, Maritza, Moullier, Philippe, Samulski, R. Jude
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10512999/
https://www.ncbi.nlm.nih.gov/pubmed/37746247
http://dx.doi.org/10.1016/j.omtm.2023.05.017

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