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Updated: Mar 22, 2026

Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice
Published on: August 2, 2018
A CRISPR Path to Engineering New Genetic Mouse Models for Cardiovascular Research
Joseph M Miano1, Qiuyu Martin Zhu2, Charles J Lowenstein2
1From the Aab Cardiovascular Research Institute, University of Rochester School of Medicine and Dentistry, Rochester, NY (J.M.M., Q.M.Z., C.J.L.); and Center for Human Genetic Research, Massachusetts General Hospital, Boston, MA (Q.M.Z.). j.m.miano@rochester.edu.
The clustered regularly interspaced short palindromic repeats (CRISPR) method simplifies genome editing in mice, enabling rapid development of cardiovascular research models. This innovation makes complex genetic modifications accessible to more laboratories worldwide.
Area of Science:
- Genetics
- Molecular Biology
- Cardiovascular Research
Background:
- Traditional mouse genome modification techniques were complex and labor-intensive.
- The clustered regularly interspaced short palindromic repeats (CRISPR) technology offers a simpler, more accessible approach.
- CRISPR has revolutionized the creation of genetically modified organisms, including mouse models.
Purpose of the Study:
- To review the history and core components of CRISPR technology.
- To provide practical guidance for CRISPR genome editing in mice.
- To highlight applications of CRISPR in cardiovascular research and discuss associated challenges.
Main Methods:
- Review of CRISPR history from prokaryotes to animal genome modification.
- Explanation of CRISPR-Cas9 system components and editing strategies (2- and 3-component).
- Summary of applications including frameshift mutations, deletions, substitutions, and tag integration.
Main Results:
- CRISPR enables efficient and versatile genome editing in mice.
- Facile generation of various genetic modifications for research purposes.
- Discussion of genotyping, mosaicism, and off-target effects.
Conclusions:
- CRISPR technology has democratized genome editing for mouse model generation.
- It accelerates research in areas like cardiovascular disease.
- Ethical considerations and clinical applications are important as the technology advances.
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