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

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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Bibliographic Details
Main Authors: Hiromi Hayashita-Kinoh, Posadas-Herrera Guillermo, Yuko Nitahara-Kasahara, Mutsuki Kuraoka, Hironori Okada, Tomoko Chiyo, Shin’ichi Takeda, Takashi Okada
Format: Article
Language:English
Published: Elsevier 2021-03-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
AAV
DMD
immune modulation
MSC
DMD dog model
microdystrophin
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050120302308

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http://www.sciencedirect.com/science/article/pii/S2329050120302308

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