A new compact adenine base editor generated through deletion of HNH and REC2 domain of SpCas9.

ABSTRACT:

Background

Adenine base editors (ABEs) are promising therapeutic gene editing tools that can efficiently convert targeted A•T to G•C base pairs in the genome. However, the large size of commonly used ABEs based on SpCas9 hinders its delivery in vivo using certain vectors such as adeno-associated virus (AAV) during preclinical applications. Despite a number of approaches having previously been attempted to overcome that challenge, including split Cas9-derived and numerous domain-deleted versions of editors, whether base editor (BE) and prime editor (PE) systems can also allow deletion of those domains remains to be proven. In this study, we present a new small ABE (sABE) with significantly reduced size.

Results

We discovered that ABE8e can tolerate large single deletions in t

SUBMITTER: Qian Y 

PROVIDER: S-EPMC10337206 | biostudies-literature | 2023 Jul

REPOSITORIES: biostudies-literature