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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...
保存先:
出版年: | Expert Opin Orphan Drugs |
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主要な著者: | , |
フォーマット: | Artigo |
言語: | Inglês |
出版事項: |
2015
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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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