Genome Editing for the Understanding and Treatment of Inherited Cardiomyopathies

被引：22
作者：
Quynh Nguyen [1 ]
Lim, Kenji Rowel Q. [1 ]
Yokota, Toshifumi [1 ,2 ]
机构：
[1] Univ Alberta, Dept Med Genet, Fac Med & Dent, Edmonton, AB T6G 2H7, Canada
[2] HM Toupin Neurol Sci Res Chair, Friends Garrett Cumming Res & Muscular Dystrophy, Edmonton, AB T6G 2H7, Canada
来源：
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES | 2020年 / 21卷 / 03期
基金：
加拿大健康研究院;
关键词：
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); PLURIPOTENT STEM-CELL; RESTORES DYSTROPHIN EXPRESSION; HYPERTROPHIC CARDIOMYOPATHY; MUSCULAR-DYSTROPHY; BARTH SYNDROME; MOUSE MODEL; CARDIAC INVOLVEMENT; CRISPR-CAS9; DUCHENNE; CAS9;
D O I：
10.3390/ijms21030733
中图分类号：
Q5 [生物化学]; Q7 [分子生物学];
学科分类号：
071010 ; 081704 ;
摘要：
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.
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页数：27
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