Therapeutic Genome Editing in Cardiovascular Diseases

被引：33

作者：

German, David M. ^{[1
]}

Mitalipov, Shoukhrat ^{[1
,2
]}

Mishra, Anusha ^{[1
]}

Kaul, Sanjiv ^{[1
]}

机构：

[1] Oregon Hlth & Sci Univ, Knight Cardiovasc Inst, UHN 62,3181 Southwest Sam Jackson Pk Rd, Portland, OR 97239 USA

[2] Oregon Hlth & Sci Univ, Ctr Embryon Cell & Gene Therapy, Portland, OR 97239 USA

来源：

JACC-BASIC TO TRANSLATIONAL SCIENCE | 2019年 / 4卷 / 01期

关键词：

CRISPR; gene editing; germline gene correction; CLONAL HEMATOPOIESIS; PCSK9; BASE; DNA; CRISPR/CAS9; MUTATIONS; NUCLEASES; ANGPTL3; MICE; CAS9;

D O I：

10.1016/j.jacbts.2018.11.004

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

A variety of genetic cardiovascular diseases may one day be curable using gene editing technology. Germline genome editing and correction promises to permanently remove monogenic cardiovascular disorders from the offspring and subsequent generations of affected families. Although technically feasible and likely to be ready for implementation in humans in the near future, this approach remains ethically controversial. Although currently beset by several technical challenges, and not yet past small animal models, somatic genome editing may also be useful for a variety of cardiovascular disorders. It potentially avoids ethical concerns about permanent editing of the germline and allows treatment of already diseased individuals. if technical challenges of Cas9-gRNA delivery (viral vector immune response, nonviral vector delivery) can be worked out, then CRISPR-Cas9 may have a significant place in the treatment of a wide variety of disorders in which partial or complete gene knockout is desired. However, CRISPR may not work for gene correction in the human heart because of tow rates of homology directed repair. Off-target effects also remain a concern, although, thus far, small animal studies have been reassuring. Some of the therapies mentioned in this review may be ready for small clinical trials in the near future. (C) 2019 The Authors. Published by Elsevier on behalf of the American College of Cardiology Foundation.

引用

页码：122 / 131

页数：10

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