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Applications of CRISPR/Cas9 for the Treatment of Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a fatal X-linked recessive neuromuscular disease prevalent in 1 in 3500 to 5000 males worldwide. As a result of mutations that interrupt the reading frame of the dystrophin gene (DMD), DMD is characterized by a loss of dystrophin protein that leads to decreased m...

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Pubblicato in:J Pers Med
Autori principali: Lim, Kenji Rowel Q., Yoon, Chantal, Yokota, Toshifumi
Natura: Artigo
Lingua:Inglês
Pubblicazione: MDPI 2018
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC6313657/
https://ncbi.nlm.nih.gov/pubmed/30477208
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/jpm8040038
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