Article

Adenine Base Editing <i>in vivo</i> with a Single Adeno-Associated Virus Vector

2021-12-13

Abstract excerpt

Base editors (BEs) have opened new avenues for the treatment of genetic diseases. However, advances in delivery approaches are needed to enable disease targeting of a broad range of tissues and cell types. Adeno-associated virus (AAV) vectors remain one of the most promising delivery vehicles for gene therapies. Currently, most BE/guide combinations and their promoters exceed the packaging limit (~5 kb) of AAVs. D...

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Literature Corpus work: e95c4056-0275-552b-b40a-48054669ed2a
DOI: 10.1101/2021.12.13.472434

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