Genome Editing and Pathological Cardiac Hypertrophy

Overview

Journal Adv Exp Med Biol

Specialties Biology
General Medicine

Date 2022 Dec 1

PMID 36454461

Authors

Takao Kato

Affiliations

Soon will be listed here.

Abstract

Three major genome editing tools, transcription activator-like effector nucleases (TALENs), zinc finger nucleases (ZFNs), and clustered regularly interspaced short palindromic repeat (CRISPR) systems, are increasingly important technologies used in the study and treatment of hereditary myocardial diseases. Germ cell genome editing and modification can permanently eliminate monogenic cardiovascular disease from the offspring of affected families and the next generation, although ethically controversial. Somatic genome editing may be a promising method for the treatment of hereditary cardiomyopathy various diseases for which gene knockout is favorable and can also treat people who are already ill, although there are currently some technical challenges. This chapter describes the application of genome editing in the experimental studies and treatment of hypertrophic cardiomyopathy as well as other cardiomyopathies.

References

Strong A, Musunuru K . Genome editing in cardiovascular diseases. Nat Rev Cardiol. 2016; 14(1):11-20. DOI: 10.1038/nrcardio.2016.139. View

Bitinaite J, Wah D, Aggarwal A, Schildkraut I . FokI dimerization is required for DNA cleavage. Proc Natl Acad Sci U S A. 1998; 95(18):10570-5. PMC: 27935. DOI: 10.1073/pnas.95.18.10570. View

Kim Y, Cha J, Chandrasegaran S . Hybrid restriction enzymes: zinc finger fusions to Fok I cleavage domain. Proc Natl Acad Sci U S A. 1996; 93(3):1156-60. PMC: 40048. DOI: 10.1073/pnas.93.3.1156. View

Urnov F, Rebar E, Holmes M, Zhang H, Gregory P . Genome editing with engineered zinc finger nucleases. Nat Rev Genet. 2010; 11(9):636-46. DOI: 10.1038/nrg2842. View

Jinek M, Chylinski K, Fonfara I, Hauer M, Doudna J, Charpentier E . A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science. 2012; 337(6096):816-21. PMC: 6286148. DOI: 10.1126/science.1225829. View

Boch J, Scholze H, Schornack S, Landgraf A, Hahn S, Kay S . Breaking the code of DNA binding specificity of TAL-type III effectors. Science. 2009; 326(5959):1509-12. DOI: 10.1126/science.1178811. View

Bogdanove A, Voytas D . TAL effectors: customizable proteins for DNA targeting. Science. 2011; 333(6051):1843-6. DOI: 10.1126/science.1204094. View

Moscou M, Bogdanove A . A simple cipher governs DNA recognition by TAL effectors. Science. 2009; 326(5959):1501. DOI: 10.1126/science.1178817. View

Christian M, cermak T, Doyle E, Schmidt C, Zhang F, Hummel A . Targeting DNA double-strand breaks with TAL effector nucleases. Genetics. 2010; 186(2):757-61. PMC: 2942870. DOI: 10.1534/genetics.110.120717. View

10.

Cong L, Ran F, Cox D, Lin S, Barretto R, Habib N . Multiplex genome engineering using CRISPR/Cas systems. Science. 2013; 339(6121):819-23. PMC: 3795411. DOI: 10.1126/science.1231143. View

11.

Mali P, Yang L, Esvelt K, Aach J, Guell M, DiCarlo J . RNA-guided human genome engineering via Cas9. Science. 2013; 339(6121):823-6. PMC: 3712628. DOI: 10.1126/science.1232033. View

12.

Hwang W, Fu Y, Reyon D, Maeder M, Tsai S, Sander J . Efficient genome editing in zebrafish using a CRISPR-Cas system. Nat Biotechnol. 2013; 31(3):227-9. PMC: 3686313. DOI: 10.1038/nbt.2501. View

13.

Cho S, Kim S, Kim J, Kim J . Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease. Nat Biotechnol. 2013; 31(3):230-2. DOI: 10.1038/nbt.2507. View

14.

Zetsche B, Gootenberg J, Abudayyeh O, Slaymaker I, Makarova K, Essletzbichler P . Cpf1 is a single RNA-guided endonuclease of a class 2 CRISPR-Cas system. Cell. 2015; 163(3):759-71. PMC: 4638220. DOI: 10.1016/j.cell.2015.09.038. View

15.

Ohiri J, McNally E . Gene Editing and Gene-Based Therapeutics for Cardiomyopathies. Heart Fail Clin. 2018; 14(2):179-188. PMC: 5849064. DOI: 10.1016/j.hfc.2017.12.006. View

16.

Nguyen Q, Lim K, Yokota T . Genome Editing for the Understanding and Treatment of Inherited Cardiomyopathies. Int J Mol Sci. 2020; 21(3). PMC: 7036815. DOI: 10.3390/ijms21030733. View

17.

German D, Mitalipov S, Mishra A, Kaul S . Therapeutic Genome Editing in Cardiovascular Diseases. JACC Basic Transl Sci. 2019; 4(1):122-131. PMC: 6390678. DOI: 10.1016/j.jacbts.2018.11.004. View

18.

Motta B, Pramstaller P, Hicks A, Rossini A . The Impact of CRISPR/Cas9 Technology on Cardiac Research: From Disease Modelling to Therapeutic Approaches. Stem Cells Int. 2018; 2017:8960236. PMC: 5757142. DOI: 10.1155/2017/8960236. View

19.

Maron B . Clinical Course and Management of Hypertrophic Cardiomyopathy. N Engl J Med. 2018; 379(7):655-668. DOI: 10.1056/NEJMra1710575. View

20.

Maron B, Towbin J, Thiene G, Antzelevitch C, Corrado D, Arnett D . Contemporary definitions and classification of the cardiomyopathies: an American Heart Association Scientific Statement from the Council on Clinical Cardiology, Heart Failure and Transplantation Committee; Quality of Care and Outcomes Research and.... Circulation. 2006; 113(14):1807-16. DOI: 10.1161/CIRCULATIONAHA.106.174287. View