Efficient precise in vivo base editing in adult dystrophic mice

Base editing is one approach used to correct mutations causing cause Duchenne muscular dystrophy (DMD), but limitations are in the requirement for a specific PAM motif and the large size beyond the packaging capacity of adeno-associated virus (AAV). Here, the authors modify the NG-targeting adenine...

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Autores principales: Li Xu, Chen Zhang, Haiwen Li, Peipei Wang, Yandi Gao, Nahush A. Mokadam, Jianjie Ma, W. David Arnold, Renzhi Han
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2021
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Acceso en línea:https://doaj.org/article/e267fd77833f4da08de183661fa8161a
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