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Progress toward Gene Therapy for Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) has been a major target for gene therapy development for nearly 30 years. DMD is among the most common genetic diseases, and isolation of the defective gene (DMD, or dystrophin) was a landmark discovery, as it was the first time a human disease gene had been cloned...

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Dettagli Bibliografici
Pubblicato in:Mol Ther
Autori principali: Chamberlain, Joel R., Chamberlain, Jeffrey S.
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society of Gene & Cell Therapy 2017
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC5417844/
https://ncbi.nlm.nih.gov/pubmed/28416280
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2017.02.019
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