In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration.

ABSTRACT: Targeted genome editing via engineered nucleases is an exciting area of biomedical research and holds potential for clinical applications. Despite rapid advances in the field, in vivo targeted transgene integration is still infeasible because current tools are inefficient, especially for non-dividing cells, which compose most adult tissues. This poses a barrier for uncovering fundamental biological principles and developing treatments for a broad range of genetic disorders. Based on clustered regularly interspaced short palindromic repeat/Cas9 (CRISPR/Cas9) technology, here we devise a homology-independent targeted integration (HITI) strategy, which allows for robust DNA knock-in in both dividing and non-dividing cells in vitro and, more importantly, in vivo (for example, in neurons of pos

SUBMITTER: Suzuki K 

PROVIDER: S-EPMC5331785 | biostudies-literature | 2016 Dec

REPOSITORIES: biostudies-literature