生物
背景(考古学)
基因组编辑
Cas9
疾病
计算生物学
遗传增强
基因
生物信息学
遗传学
清脆的
医学
病理
古生物学
作者
Tadahiro Tadokoro,Eric N. Olson,Ning Liu
标识
DOI:10.1146/annurev-genet-011725-094039
摘要
Cardiovascular disease is the leading cause of global morbidity and mortality, despite advances in pharmacological and surgical interventions. The emergence of CRISPR-Cas9 genome editing technology offers promising approaches for correcting genetic causes of hereditary cardiovascular disorders and modulating pathogenic signaling pathways implicated in various heart diseases. However, several challenges with respect to in vivo delivery of gene editing components, as well as important safety considerations, remain to be addressed in the path toward possible clinical application. We review current gene editing strategies, their potential therapeutic applications in the context of a variety of cardiovascular disorders, and their respective merits, limitations, and regulatory considerations. The rapid advances in this field combined with the many opportunities for deploying gene editing therapies for cardiovascular disorders augur well for the future of this transformative technology.
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