» Articles » PMID: 27609628

Genome Editing in Cardiovascular Diseases

Overview
Journal Nat Rev Cardiol
Date 2016 Sep 10
PMID 27609628
Citations 47
Authors
Affiliations
Soon will be listed here.
Abstract

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.

Citing Articles

Rapid Detection of Fusions in Acute Promyelocytic Leukemia: CRISPR/Cas9 Nanopore Sequencing with Adaptive Sampling.

Middlezong W, Stinnett V, Phan M, Phan B, Morsberger L, Klausner M Biomolecules. 2025; 14(12.

PMID: 39766302 PMC: 11674480. DOI: 10.3390/biom14121595.


Cardiomyopathy: pathogenesis and therapeutic interventions.

Huang S, Li J, Li Q, Wang Q, Zhou X, Chen J MedComm (2020). 2024; 5(11):e772.

PMID: 39465141 PMC: 11502724. DOI: 10.1002/mco2.772.


Application and perspective of CRISPR/Cas9 genome editing technology in human diseases modeling and gene therapy.

Zhang M, Li H, Jin Y Front Genet. 2024; 15:1364742.

PMID: 38666293 PMC: 11043577. DOI: 10.3389/fgene.2024.1364742.


Current Landscape of Gene Therapy for the Treatment of Cardiovascular Disorders.

Wal P, Aziz N, Singh C, Rasheed A, Tyagi L, Agrawal A Curr Gene Ther. 2024; 24(5):356-376.

PMID: 38288826 DOI: 10.2174/0115665232268840231222035423.


Innovative Strategies of Reprogramming Immune System Cells by Targeting CRISPR/Cas9-Based Genome-Editing Tools: A New Era of Cancer Management.

Allemailem K, Alsahli M, Almatroudi A, Alrumaihi F, Al Abdulmonem W, Moawad A Int J Nanomedicine. 2023; 18:5531-5559.

PMID: 37795042 PMC: 10547015. DOI: 10.2147/IJN.S424872.


References
1.
Coelho T, Adams D, Silva A, Lozeron P, Hawkins P, Mant T . Safety and efficacy of RNAi therapy for transthyretin amyloidosis. N Engl J Med. 2013; 369(9):819-29. DOI: 10.1056/NEJMoa1208760. View

2.
Lepperhof V, Polchynski O, Kruttwig K, Bruggemann C, Neef K, Drey F . Bioluminescent imaging of genetically selected induced pluripotent stem cell-derived cardiomyocytes after transplantation into infarcted heart of syngeneic recipients. PLoS One. 2014; 9(9):e107363. PMC: 4167328. DOI: 10.1371/journal.pone.0107363. View

3.
Zhang M, DAniello C, Verkerk A, Wrobel E, Frank S, Ward-van Oostwaard D . Recessive cardiac phenotypes in induced pluripotent stem cell models of Jervell and Lange-Nielsen syndrome: disease mechanisms and pharmacological rescue. Proc Natl Acad Sci U S A. 2014; 111(50):E5383-92. PMC: 4273331. DOI: 10.1073/pnas.1419553111. View

4.
Orthwein A, Noordermeer S, Wilson M, Landry S, Enchev R, Sherker A . A mechanism for the suppression of homologous recombination in G1 cells. Nature. 2015; 528(7582):422-6. PMC: 4880051. DOI: 10.1038/nature16142. View

5.
Branzei D, Foiani M . Regulation of DNA repair throughout the cell cycle. Nat Rev Mol Cell Biol. 2008; 9(4):297-308. DOI: 10.1038/nrm2351. View