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Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: Current Status of Therapeutic Approaches

Duchenne muscular dystrophy (DMD), a rare genetic disorder characterized by progressive muscle weakness, is caused by the absence or a decreased amount of the muscle cytoskeletal protein dystrophin. Currently, several therapeutic approaches to cure DMD are being investigated, which can be categorize...

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Pubblicato in:J Pers Med
Autori principali: Shimizu-Motohashi, Yuko, Komaki, Hirofumi, Motohashi, Norio, Takeda, Shin’ichi, Yokota, Toshifumi, Aoki, Yoshitsugu
Natura: Artigo
Lingua:Inglês
Pubblicazione: MDPI 2019
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC6462907/
https://ncbi.nlm.nih.gov/pubmed/30621068
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/jpm9010001
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