Genome editing in cardiovascular diseases.
Genome-editing tools, which include zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated 9 (Cas9) systems, have emerged as an invaluable technology to achieve somatic and germline genomic manipulation in cells and model organisms for multiple applications, including the creation of knockout alleles, introducing desired mutations into genomic DNA, and inserting novel transgenes. Genome editing is being rapidly adopted into all fields of biomedical research, including the cardiovascular field, where it has facilitated a greater understanding of lipid metabolism, electrophysiology, cardiomyopathies, and other cardiovascular disorders, has helped to create a wider variety of cellular and animal models, and has opened the door to a new class of therapies. In this Review, we discuss the applications of genome-editing technology throughout cardiovascular disease research and the prospect of in vivo genome-editing therapies in the future. We also describe some of the existing limitations of genome-editing tools that will need to be addressed if cardiovascular genome editing is to achieve its full scientific and therapeutic potential.
Department of Pediatrics, St. Christopher's Hospital for Children, 160 East Erie Avenue, Philadelphia, Pennsylvania 19134, USA.|Cardiovascular Institute, Department of Medicine, and Department of Genetics, Perelman School of Medicine at the University of Pennsylvania, 11-136 Smilow Center for Translational Research, 3400 Civic Center Boulevard, Building 421, Philadelphia, Pennsylvania 19104, USA.
Alanna
A
Strong
Cardiovascular Institute, Department of Medicine, and Department of Genetics, Perelman School of Medicine at the University of Pennsylvania, 11-136 Smilow Center for Translational Research, 3400 Civic Center Boulevard, Building 421, Philadelphia, Pennsylvania 19104, USA.
Kiran
K
Musunuru
D002318:Cardiovascular Diseases / Q000235:genetics*; D000072669:Gene Editing; D006801:Humans