Improved transduction of canine X-linked muscular dystrophy with rAAV9-microdystrophin via multipotent MSC pretreatment

Duchenne muscular dystrophy (DMD) is a severe congenital disease associated with mutation of the dystrophin gene. Supplementation of dystrophin using recombinant adeno-associated virus (rAAV) has promise as a treatment for DMD, although vector-related general toxicities, such as liver injury, neurot...

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Detalles Bibliográficos
Autores principales: Hayashita-Kinoh, Hiromi, Guillermo, Posadas-Herrera, Nitahara-Kasahara, Yuko, Kuraoka, Mutsuki, Okada, Hironori, Chiyo, Tomoko, Takeda, Shin’ichi, Okada, Takashi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7773564/
https://www.ncbi.nlm.nih.gov/pubmed/33426145
http://dx.doi.org/10.1016/j.omtm.2020.11.003

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