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Gene Therapy for Duchenne muscular dystrophy

INTRODUCTION: Duchenne muscular dystrophy (DMD) is a relatively common inherited disorder caused by defective expression of the protein dystrophin. The most direct approach to treating this disease would be to restore dystrophin production in muscle. Recent progress has greatly increased the prospec...

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Détails bibliographiques
Publié dans:Expert Opin Orphan Drugs
Auteurs principaux: Ramos, Julian, Chamberlain, Jeffrey S
Format: Artigo
Langue:Inglês
Publié: 2015
Sujets:
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC4651452/
https://ncbi.nlm.nih.gov/pubmed/26594599
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1517/21678707.2015.1088780
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