Ontology highlight
ABSTRACT:
SUBMITTER: Zheng C
PROVIDER: S-EPMC8899602 | biostudies-literature | 2022 Mar
REPOSITORIES: biostudies-literature
Molecular therapy : the journal of the American Society of Gene Therapy 20220105 3
Prime editor (PE) has tremendous promise for gene therapy. However, it remains a challenge to deliver PE (>6.3 kb) in vivo. Although PE can be split into two fragments and delivered using dual adeno-associated viruses (AAVs), choice of split sites within Cas9-which affects editing efficiency-is limited due to the large size of PE. Furthermore, overexpressing reverse transcriptase in mammalian cells might disrupt translation termination via its RNase H domain. Here, we developed a compact PE with ...[more]