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Article

Efficient precise <i>in vivo</i> base editing in adult dystrophic mice

2020-06-26

Abstract excerpt

<h4>ABSTRACT</h4> <h4>Background</h4> Recent advances in the base editing technology 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) sequence within a suitable window near the mutation site...

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Literature Corpus work
5a39b4bd-c736-5fbd-9d72-6beaaa9a075b
DOI
10.1101/2020.06.24.169292
Open publication

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Efficient precise <i>in vivo</i> base editing in adult dystrophic miceDOI 10.1101/2020.06.24.169292
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