A Fluorescent Reporter Mouse for In Vivo Assessment of Genome Editing with Diverse Cas Nucleases and Prime Editors.

Autor: Chen Z; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA., Kwan SY; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA., Mir A; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA., Hazeltine M; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA., Shin M; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA., Liang SQ; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA., Chan IL; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA., Kelly K; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA., Ghanta KS; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA., Gaston N; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA., Cao Y; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA., Xie J; Li Weibo Institute for Rare Diseases Research, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA.; NeuroNexus Institute, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA.; Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA.; Viral Vector Core, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA.; Department of Microbiology and Physiological Systems, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA., Gao G; Li Weibo Institute for Rare Diseases Research, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA.; NeuroNexus Institute, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA.; Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA.; Viral Vector Core, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA.; Department of Microbiology and Physiological Systems, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA., Xue W; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA.; Li Weibo Institute for Rare Diseases Research, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA.; Program in Molecular Medicine, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA.; Department of Molecular, Cell and Cancer Biology, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA., Sontheimer EJ; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA.; Li Weibo Institute for Rare Diseases Research, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA.; Program in Molecular Medicine, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA.; Department of Biochemistry and Molecular Biotechnology, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA., Watts JK; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA.; Li Weibo Institute for Rare Diseases Research, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA.; NeuroNexus Institute, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA.; Department of Biochemistry and Molecular Biotechnology, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA.
Jazyk: angličtina
Zdroj: The CRISPR journal [CRISPR J] 2023 Dec; Vol. 6 (6), pp. 570-582.
DOI: 10.1089/crispr.2023.0048
Abstrakt: CRISPR-based genome-editing technologies, including nuclease editing, base editing, and prime editing, have recently revolutionized the development of therapeutics targeting disease-causing mutations. To advance the assessment and development of genome editing tools, a robust mouse model is valuable, particularly for evaluating in vivo activity and delivery strategies. In this study, we successfully generated a knock-in mouse line carrying the Traffic Light Reporter design known as TLR-multi-Cas variant 1 (TLR-MCV1). We comprehensively validated the functionality of this mouse model for both in vitro and in vivo nuclease and prime editing. The TLR-MCV1 reporter mouse represents a versatile and powerful tool for expediting the development of editing technologies and their therapeutic applications.
Databáze: MEDLINE