Genome Editing and Myocardial Development

Sifa Turan; J Richard Chaillet; Margaret C Stapleton; Yijen L Wu

doi:10.1007/978-981-19-5642-3_4

Genome Editing and Myocardial Development

Adv Exp Med Biol. 2023:1396:53-73. doi: 10.1007/978-981-19-5642-3_4.

Authors

Sifa Turan¹, J Richard Chaillet^{1

2}, Margaret C Stapleton^{3

4}, Yijen L Wu^{5

6}

Affiliations

¹ Department of Obstetrics, Gynecology and Reproductive Sciences, University of Maryland School of Medicine, Baltimore, MD, USA.
² Magee-Womens Research Institute, School of Medicine, University of Pittsburgh, Pittsburgh, PA, USA.
³ Department of Developmental Biology, School of Medicine, University of Pittsburgh, Pittsburgh, PA, USA.
⁴ Rangos Research Center Animal Imaging Core, Children's Hospital of Pittsburgh of UPMC, School of Medicine, University of Pittsburgh, Pittsburgh, PA, USA.
⁵ Department of Developmental Biology, School of Medicine, University of Pittsburgh, Pittsburgh, PA, USA. yijenwu@pitt.edu.
⁶ Rangos Research Center Animal Imaging Core, Children's Hospital of Pittsburgh of UPMC, School of Medicine, University of Pittsburgh, Pittsburgh, PA, USA. yijenwu@pitt.edu.

PMID: 36454459
DOI: 10.1007/978-981-19-5642-3_4

Abstract

Congenital heart disease (CHD) has a strong genetic etiology, making it a likely candidate for therapeutic intervention using genetic editing. Complex genetics involving an orchestrated series of genetic events and over 400 genes are responsible for myocardial development. Cooperation is required from a vast series of genetic networks, and mutations in such can lead to CHD and cardiovascular abnormalities, affecting up to 1% of all live births. Genome editing technologies are becoming better studied and with time and improved logistics, CHD could be a prime therapeutic target. Syndromic, nonsyndromic, and cases of familial inheritance all involve identifiable causative mutations and thus have the potential for genome editing therapy. Mouse models are well-suited to study and predict clinical outcome. This review summarizes the anatomical and genetic timeline of myocardial development in both mice and humans, the potential of gene editing in typical CHD categories, as well as the use of mice thus far in reproducing models of human CHD and correcting the mutations that create them.

Keywords: Cardiovascular disease; Congenital heart disease (CHD); Heart development.

Publication types

Review

MeSH terms

Animals
Disease Models, Animal
Gene Editing*
Gene Regulatory Networks*
Humans
Inheritance Patterns
Mice
Mutation

Grants and funding

R21 EB023507/EB/NIBIB NIH HHS/United States