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Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy
Duchenne muscular dystrophy is one of the most common inherited genetic diseases and is caused by mutations to the DMD gene that encodes the dystrophin protein. Recent advances in genome editing and gene therapy offer hope for the development of potential therapeutics. Truncated versions of the DMD...
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| Publicado no: | Hum Genet |
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| Main Authors: | , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2016
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5006996/ https://ncbi.nlm.nih.gov/pubmed/27542949 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s00439-016-1725-z |
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