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Progress toward Gene Therapy for Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) has been a major target for gene therapy development for nearly 30 years. DMD is among the most common genetic diseases, and isolation of the defective gene (DMD, or dystrophin) was a landmark discovery, as it was the first time a human disease gene had been cloned...
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| Publicado no: | Mol Ther |
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| Main Authors: | , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
American Society of Gene & Cell Therapy
2017
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5417844/ https://ncbi.nlm.nih.gov/pubmed/28416280 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2017.02.019 |
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