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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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Dades bibliogràfiques
Publicat a:Expert Opin Orphan Drugs
Autors principals: Ramos, Julian, Chamberlain, Jeffrey S
Format: Artigo
Idioma:Inglês
Publicat: 2015
Matèries:
Accés en línia: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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