Article
Efficient precise in vivo base editing in adult dystrophic mice.
Nature communications - 17 Jun 2021
Xu Li, Zhang Chen, Li Haiwen, Wang Peipei, Gao Yandi, Mokadam Nahush A, Ma Jianjie, Arnold W David, Han Renzhi
Abstract excerpt
Recent advances in base editing have created an exciting opportunity to precisely correct disease-causing mutations. However, the large size of base editors and their inherited off-target activities pose challenges for in vivo base editing. Moreover, the requirement of a protospacer adjacent motif (PAM) nearby the mutation site further limits the targeting feasibility. Here we modify the NG-targeting adenine base...
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