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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Bibliographic Details
Main Authors: Li Xu, Chen Zhang, Haiwen Li, Peipei Wang, Yandi Gao, Nahush A. Mokadam, Jianjie Ma, W. David Arnold, Renzhi Han
Format: Article
Language:English
Published: Nature Publishing Group 2021-06-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-021-23996-y