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Genome Editing Gene Therapy for Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a severe genetic disorder caused by loss of function of the dystrophin gene on the X chromosome. Gene augmentation of dystrophin is challenging due to the large size of the dystrophin cDNA. Emerging genome editing technologies, such as TALEN and CRISPR-Cas9 syste...
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| Publicado no: | J Neuromuscul Dis |
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| Autor principal: | |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
IOS Press
2015
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5240599/ https://ncbi.nlm.nih.gov/pubmed/27858753 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3233/JND-150116 |
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