Genome Editing for the Understanding and Treatment of Cardiomyopathies

Quynh Nguyen; Kenji Rowel Q. Lim; Toshifumi Yokota

doi:10.20944/preprints201912.0385.v1

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preprints.org > medicine and pharmacology > cardiac and cardiovascular systems > doi: 10.20944/preprints201912.0385.v1

Preprint Review Version 1 Preserved in Portico This version is not peer-reviewed

Genome Editing for the Understanding and Treatment of Cardiomyopathies

Quynh Nguyen

Kenji Rowel Q. Lim

Toshifumi Yokota

Version 1 : Received: 28 December 2019 / Approved: 29 December 2019 / Online: 29 December 2019 (13:41:48 CET)

A peer-reviewed article of this Preprint also exists.

Nguyen, Q.; Lim, K.R.Q.; Yokota, T. Genome Editing for the Understanding and Treatment of Inherited Cardiomyopathies. Int. J. Mol. Sci. 2020, 21, 733. Nguyen, Q.; Lim, K.R.Q.; Yokota, T. Genome Editing for the Understanding and Treatment of Inherited Cardiomyopathies. Int. J. Mol. Sci. 2020, 21, 733.

Abstract

Cardiomyopathies are diseases of heart muscle, a significant percentage of which are genetic in origin. Cardiomyopathies can be classified as dilated, hypertrophic, restrictive, arrhythmogenic right ventricular or left ventricular non-compaction, although mixed morphologies are possible. A subset of neuromuscular disorders, notably Duchenne and Becker muscular dystrophies, are also characterized by cardiomyopathy aside from skeletal myopathy. The global burden of cardiomyopathies is certainly high, necessitating further research and novel therapies. Genome editing tools, which include zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs) and clustered regularly interspaced short palindromic repeats (CRISPR) systems have emerged as increasingly important technologies in studying this group of cardiovascular disorders. In this review, we discuss the applications of genome editing in the understanding and treatment of cardiomyopathy. We also describe recent advances in genome editing that may help improve these applications, and some future prospects for genome editing in cardiomyopathy treatment.

Keywords

dilated cardiomyopathy (DCM); hypertrophic cardiomyopathy (HCM); restrictive cardiomyopathy (RCM); arrhythmogenic right ventricular cardiomyopathy (ARVC); left ventricular non-compaction cardiomyopathy (LVNC); Duchenne muscular dystrophy; dystrophin; genome editing; CRISPR/Cas9; Cpf1 (Cas12a)

Subject

Medicine and Pharmacology, Cardiac and Cardiovascular Systems

Copyright: This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.

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Comments (1)

Comment 1

Received: 10 January 2020
Commenter: Ian M MacDonald
The commenter has declared there is no conflict of interests.

Comment: This article provides useful insights into current thinking about gene therapies in the cardiomyopathies and heritable disorders.

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