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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Bibliografski detalji
Izdano u:Mol Ther
Glavni autori: Ramos, Julian N., Hollinger, Katrin, Bengtsson, Niclas E., Allen, James M., Hauschka, Stephen D., Chamberlain, Jeffrey S.
Format: Artigo
Jezik:Inglês
Izdano: American Society of Gene & Cell Therapy 2019
Teme:
Original Article
Online pristup: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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