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CRISPR/Cas9 gene-editing strategies in cardiovascular cells.
Cardiovasc Res. 2020 04 01; 116(5):894-907.CR

Abstract

Cardiovascular diseases are among the main causes of morbidity and mortality in Western countries and considered as a leading public health issue. Therefore, there is a strong need for new disease models to support the development of novel therapeutics approaches. The successive improvement of genome editing tools with zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and more recently with clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated 9 (Cas9) has enabled the generation of genetically modified cells and organisms with much greater efficiency and precision than before. The simplicity of CRISPR/Cas9 technology made it especially suited for different studies, both in vitro and in vivo, and has been used in multiple studies evaluating gene functions, disease modelling, transcriptional regulation, and testing of novel therapeutic approaches. Notably, with the parallel development of human induced pluripotent stem cells (hiPSCs), the generation of knock-out and knock-in human cell lines significantly increased our understanding of mutation impacts and physiopathological mechanisms within the cardiovascular domain. Here, we review the recent development of CRISPR-Cas9 genome editing, the alternative tools, the available strategies to conduct genome editing in cardiovascular cells with a focus on its use for correcting mutations in vitro and in vivo both in germ and somatic cells. We will also highlight that, despite its potential, CRISPR/Cas9 technology comes with important technical and ethical limitations. The development of CRISPR/Cas9 genome editing for cardiovascular diseases indeed requires to develop a specific strategy in order to optimize the design of the genome editing tools, the manipulation of DNA repair mechanisms, the packaging and delivery of the tools to the studied organism, and the assessment of their efficiency and safety.

Authors+Show Affiliations

Paris Cardiovascular Research Center PARCC, Université de Paris, INSERM, 56 Rue Leblanc, 75015 Paris, France.Paris Cardiovascular Research Center PARCC, Université de Paris, INSERM, 56 Rue Leblanc, 75015 Paris, France.Paris Cardiovascular Research Center PARCC, Université de Paris, INSERM, 56 Rue Leblanc, 75015 Paris, France. Centre d'Investigations Cliniques CIC1418, AP-HP, Hôpital Européen Georges Pompidou, 75015 Paris, France.

Pub Type(s)

Journal Article
Research Support, Non-U.S. Gov't
Review

Language

eng

PubMed ID

31584620

Citation

Vermersch, Eva, et al. "CRISPR/Cas9 Gene-editing Strategies in Cardiovascular Cells." Cardiovascular Research, vol. 116, no. 5, 2020, pp. 894-907.
Vermersch E, Jouve C, Hulot JS. CRISPR/Cas9 gene-editing strategies in cardiovascular cells. Cardiovasc Res. 2020;116(5):894-907.
Vermersch, E., Jouve, C., & Hulot, J. S. (2020). CRISPR/Cas9 gene-editing strategies in cardiovascular cells. Cardiovascular Research, 116(5), 894-907. https://doi.org/10.1093/cvr/cvz250
Vermersch E, Jouve C, Hulot JS. CRISPR/Cas9 Gene-editing Strategies in Cardiovascular Cells. Cardiovasc Res. 2020 04 1;116(5):894-907. PubMed PMID: 31584620.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - CRISPR/Cas9 gene-editing strategies in cardiovascular cells. AU - Vermersch,Eva, AU - Jouve,Charlène, AU - Hulot,Jean-Sébastien, PY - 2018/12/12/received PY - 2019/05/05/revised PY - 2019/09/26/accepted PY - 2019/10/5/pubmed PY - 2020/10/21/medline PY - 2019/10/5/entrez KW - CRISPR/Cas9 KW - Cardiomyopathy KW - Disease modelling KW - Genetics KW - Genome editing SP - 894 EP - 907 JF - Cardiovascular research JO - Cardiovasc Res VL - 116 IS - 5 N2 - Cardiovascular diseases are among the main causes of morbidity and mortality in Western countries and considered as a leading public health issue. Therefore, there is a strong need for new disease models to support the development of novel therapeutics approaches. The successive improvement of genome editing tools with zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and more recently with clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated 9 (Cas9) has enabled the generation of genetically modified cells and organisms with much greater efficiency and precision than before. The simplicity of CRISPR/Cas9 technology made it especially suited for different studies, both in vitro and in vivo, and has been used in multiple studies evaluating gene functions, disease modelling, transcriptional regulation, and testing of novel therapeutic approaches. Notably, with the parallel development of human induced pluripotent stem cells (hiPSCs), the generation of knock-out and knock-in human cell lines significantly increased our understanding of mutation impacts and physiopathological mechanisms within the cardiovascular domain. Here, we review the recent development of CRISPR-Cas9 genome editing, the alternative tools, the available strategies to conduct genome editing in cardiovascular cells with a focus on its use for correcting mutations in vitro and in vivo both in germ and somatic cells. We will also highlight that, despite its potential, CRISPR/Cas9 technology comes with important technical and ethical limitations. The development of CRISPR/Cas9 genome editing for cardiovascular diseases indeed requires to develop a specific strategy in order to optimize the design of the genome editing tools, the manipulation of DNA repair mechanisms, the packaging and delivery of the tools to the studied organism, and the assessment of their efficiency and safety. SN - 1755-3245 UR - https://www.unboundmedicine.com/medline/citation/31584620/CRISPR/Cas9_gene_editing_strategies_in_cardiovascular_cells_ L2 - https://academic.oup.com/cardiovascres/article-lookup/doi/10.1093/cvr/cvz250 DB - PRIME DP - Unbound Medicine ER -