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Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a lethal muscle disease caused by dystrophin gene mutation. Conceptually, replacing the mutated gene with a normal one would cure the disease. However, this task has encountered significant challenges due to the enormous size of the gene and the distribution of m...
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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American Society of Gene & Cell Therapy
2018
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6171037/ https://www.ncbi.nlm.nih.gov/pubmed/30093306 http://dx.doi.org/10.1016/j.ymthe.2018.07.011 |