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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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Publicado en:Expert Opin Orphan Drugs
Autores principales: Ramos, Julian, Chamberlain, Jeffrey S
Formato: Artigo
Lenguaje:Inglês
Publicado: 2015
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Acceso en línea: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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