In Vivo Genome Editing Restores Dystrophin Expression and Cardiac Function in Dystrophic Mice
Rationale: Duchenne muscular dystrophy is a severe inherited form of muscular dystrophy caused by mutations in the reading frame of the dystrophin gene disrupting its protein expression. Dystrophic cardiomyopathy is a leading cause of death in Duchenne muscular dystrophy patients, and currently no e...
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| Main Authors: | , , , , , , , , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2017
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| Acesso em linha: | https://doi.org/10.1161/circresaha.117.310996 https://www.ahajournals.org/doi/pdf/10.1161/CIRCRESAHA.117.310996 |
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