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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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| Veröffentlicht in: | Expert Opin Orphan Drugs |
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| Hauptverfasser: | , |
| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
2015
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| Schlagworte: | |
| Online Zugang: | 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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