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Duchenne muscular dystrophy gene therapy: Lost in translation?
A milestone of molecular medicine is the identification of dystrophin gene mutation as the cause of Duchenne muscular dystrophy (DMD). Over the last 2 decades, major advances in dystrophin biology and gene delivery technology have created an opportunity to treat DMD with gene therapy. Remarkable suc...
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2011
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| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3117615/ https://ncbi.nlm.nih.gov/pubmed/21691429 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.2147/RRB.S13463 |
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