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In Vivo Genome Editing Restores Dystrophin Expression and Cardiac Function in Dystrophic Mice
RATIONALE: Duchenne muscular dystrophy (DMD) 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 DMD patients and currently no effective treatment...
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| Publicado no: | Circ Res |
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| Main Authors: | , , , , , , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2017
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5623072/ https://ncbi.nlm.nih.gov/pubmed/28790199 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1161/CIRCRESAHA.117.310996 |
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