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Dystrophin Gene-Editing Stability Is Dependent on Dystrophin Levels in Skeletal but Not Cardiac Muscles
Gene editing is often touted as a permanent method for correcting mutations, but its long-term benefits in Duchenne muscular dystrophy (DMD) may depend on sufficiently high editing efficiencies to halt muscle degeneration. Here, we explored the persistence of dystrophin expression following recombin...
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| Pubblicato in: | Mol Ther |
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| Autori principali: | , , , |
| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
American Society of Gene & Cell Therapy
2021
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| Soggetti: | |
| Accesso online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7934576/ https://ncbi.nlm.nih.gov/pubmed/33160075 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2020.11.003 |
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