Application of CRISPR-Cas9 gene editing for congenital heart disease.
Application of CRISPR-Cas9 gene editing for congenital heart disease.
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CRISPR-CAS9基因编辑的应用用于先天性心脏病。
DOI：
10.3345/cep.2020.02096
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发表时间：
2021-06
影响因子：
4.2
通讯作者：
Wang DZ
中科院分区：
文献类型：
--
作者：
Seok H;Deng R;Cowan DB;Wang DZ
规则间隔短回文重复序列和CRISPR相关蛋白9（CRISPR-Cas9）是一种古老的原核防御系统，它在少量指导RNA的控制下精确切割外源基因组DNA。CRISPR-Cas9系统有助于有效的双链DNA切割，最近已被用于基因组编辑，以创建或纠正导致疾病的遗传基因突变。先天性心脏病（CHD）通常由基因突变引起，如碱基替换、缺失和插入，这些突变导致多种发育缺陷，并且仍然是出生缺陷的主要原因。儿童CHD患者表现出一系列心脏异常，如间隔缺损、瓣膜缺损和异常腔发育。CHD发病于产前，常导致儿童早期死亡。由于基于CRISPR-Cas9的基因组编辑技术因其预防和治疗疾病的潜力而获得了相当大的关注，我们将回顾CRISPR-Cas9系统作为基因组编辑工具，并重点关注其在冠心病治疗中的应用。
Clustered regularly interspaced short palindromic repeats and CRISPR-associated protein 9 (CRISPR-Cas9) is an ancient prokaryotic defense system that precisely cuts foreign genomic DNA under the control of a small number of guide RNAs. The CRISPR-Cas9 system facilitates efficient double-stranded DNA cleavage that has been recently adopted for genome editing to create or correct inherited genetic mutations causing disease. Congenital heart disease (CHD) is generally caused by genetic mutations such as base substitutions, deletions, and insertions, which result in diverse developmental defects and remains a leading cause of birth defects. Pediatric CHD patients exhibit a spectrum of cardiac abnormalities such as septal defects, valvular defects, and abnormal chamber development. CHD onset occurs during the prenatal period and often results in early lethality during childhood. Because CRISPR-Cas9-based genome editing technology has gained considerable attention for its potential to prevent and treat diseases, we will review the CRISPR-Cas9 system as a genome editing tool and focus on its therapeutic application for CHD.
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