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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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Detalhes bibliográficos
Publicado no:Expert Opin Orphan Drugs
Main Authors: Ramos, Julian, Chamberlain, Jeffrey S
Formato: Artigo
Idioma:Inglês
Publicado em: 2015
Assuntos:
Acesso em linha: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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