Variable rescue of microtubule and physiological phenotypes in mdx muscle expressing different miniaturized dystrophins

Delivery of miniaturized dystrophin genes via adeno-associated viral vectors is one leading approach in development to treat Duchenne muscular dystrophy. Here we directly compared the functionality of five mini- and micro-dystrophins via skeletal muscle-specific transgenic expression in dystrophin-d...

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Bibliographic Details
Published in:Hum Mol Genet
Main Authors: Nelson, D’anna M, Lindsay, Angus, Judge, Luke M, Duan, Dongsheng, Chamberlain, Jeffrey S, Lowe, Dawn A, Ervasti, James M
Format: Artigo
Language:Inglês
Published: Oxford University Press 2018
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Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC5985723/
https://ncbi.nlm.nih.gov/pubmed/29618008
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddy113
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