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BIN1 Modulation in Vivo Rescues Dynamin-Related Myopathy.

Valentina Maria Lionello,Christine Kretz,Evelina Edelweiss,Corinne Crucifix,Raquel Gomez-Oca,Nadia Messaddeq,Suzie Buono,Pascale Koebel,Xenia Massana Munoz,Nadege Diedhiou,Belinda S. Cowling, Marc Bitoun,Jocelyn Laporte

Proceedings of the National Academy of Sciences of the United States of America（2022）

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关键词
congenital myopathy,membrane remodeling,gene therapy,dynamin,amphiphysin
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