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Development of Novel Micro-dystrophins with Enhanced Functionality
Gene therapies using adeno-associated viral (AAV) vectors have advanced into clinical trials for several diseases, including Duchenne muscular dystrophy (DMD). A limitation of AAV is the carrying capacity (∼5 kb) available for genes and regulatory cassettes (RCs). These size constraints are problema...
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| Publié dans: | Mol Ther |
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| Auteurs principaux: | , , , , , |
| Format: | Artigo |
| Langue: | Inglês |
| Publié: |
American Society of Gene & Cell Therapy
2019
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| Sujets: | |
| Accès en ligne: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6403485/ https://ncbi.nlm.nih.gov/pubmed/30718090 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2019.01.002 |
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