Novel reporter mouse models useful for evaluating in vivo gene editing and for optimization of methods of delivering genome editing tools.

ABSTRACT: The clustered regularly interspersed palindromic repeats (CRISPR) system is a powerful genome-editing tool to modify genomes, virtually in any species. The CRISPR tool has now been utilized in many areas of medical research, including gene therapy. Although several proof-of-concept studies show the feasibility of in vivo gene therapy applications for correcting disease-causing mutations, and new and improved tools are constantly being developed, there are not many choices of suitable reporter models to evaluate genome editor tools and their delivery methods. Here, we developed and validated reporter mouse models containing a single copy of disrupted EGFP (ΔEGFP) via frameshift mutations. We tested several delivery methods for validation of the reporters, and we demonstrated t

SUBMITTER: Miura H 

PROVIDER: S-EPMC8020343 | biostudies-literature | 2021 Jun

REPOSITORIES: biostudies-literature