Article
Allele-Specific Suppression of Variant MHC With High-Precision RNA Nuclease CRISPR-Cas13d Prevents Hypertrophic Cardiomyopathy.
Circulation - 23 Jul 2024
Yang Ping, Lou Yingmei, Geng Zilong, Guo Zhizhao, Wu Shuo, Li Yige, Song Kaiyuan, Shi Ting, Zhang Shasha, Xiong Junhao, Chen Alex F, Li Dali, Pu William T, Da Lintai, Zhang Yan, Sun Kun, Zhang Bing
Abstract excerpt
BACKGROUND: Familial hypertrophic cardiomyopathy has severe clinical complications of heart failure, arrhythmia, and sudden cardiac death. Heterozygous single nucleotide variants (SNVs) of sarcomere genes such as MYH7 are the leading cause of this type of disease. CRISPR-Cas13 (clustered regularly interspaced short palindromic repeats and their associated protein 13) is an emerging gene therapy approach for...
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