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CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice
Duchenne muscular dystrophy (DMD) is a degenerative muscle disease caused by genetic mutations that lead to the disruption of dystrophin in muscle fibers. There is no curative treatment for this devastating disease. Clustered regularly interspaced short palindromic repeat/Cas9 (CRISPR/Cas9) has emer...
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| Опубликовано в: : | Mol Ther |
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| Главные авторы: | , , , , , , , , |
| Формат: | Artigo |
| Язык: | Inglês |
| Опубликовано: |
Nature Publishing Group
2016
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| Предметы: | |
| Online-ссылка: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4786912/ https://ncbi.nlm.nih.gov/pubmed/26449883 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2015.192 |
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