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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...

詳細記述

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書誌詳細
出版年:Expert Opin Orphan Drugs
主要な著者: Ramos, Julian, Chamberlain, Jeffrey S
フォーマット: Artigo
言語:Inglês
出版事項: 2015
主題:
オンライン・アクセス: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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