400. Improved Transduction of Canine X-Linked Muscular Dystrophy With rAAV9-Microdystrophin By Using MSCs Pretreatment

Hiromi Hayashita-Kinoh,Hironori Okada,Yuko Nitahara-Kasahara,Tomoko Chiyo,Kiwamu Imagawa,Katsuhiko Tachibana,Shin'ichi Takeda,Takashi Okada

MOLECULAR THERAPY（2015）

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摘要

Duchenne muscular dystrophy (DMD) is a congenital disease causing progressive deterioration of skeletal and cardiac muscles because of mutations in the dystrophin gene. Supplementation of dystrophin using rAAV is effective to improve pathogenesis of animal models of DMD. However, we have previously reported that local injection of rAAV2 or rAAV8 to canine

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关键词

AAV,DMD,immune modulation,MSC,DMD dog model,microdystrophin

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