AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice

CRISPR editing of muscle stem cells (MuSCs) with adeno-associated virus serotype-9 (AAV9) holds promise for sustained gene repair therapy for muscular dystrophies. However, conflicting evidence exists on whether AAV9 transduces MuSCs. To rigorously address this question, we used a muscle graft model...

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Publicat a:Mol Ther
Autors principals: Nance, Michael E., Shi, Ruicheng, Hakim, Chady H., Wasala, Nalinda B., Yue, Yongping, Pan, Xiufang, Zhang, Tracy, Robinson, Carolyn A., Duan, Sean X., Yao, Gang, Yang, N. Nora, Chen, Shi-jie, Wagner, Kathryn R., Gersbach, Charles A., Duan, Dongsheng
Format: Artigo
Idioma:Inglês
Publicat: American Society of Gene & Cell Therapy 2019
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Original Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC6731180/
https://ncbi.nlm.nih.gov/pubmed/31327755
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2019.06.012
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