Article
Genome editing with CRISPR/Cas9 in postnatal mice corrects PRKAG2 cardiac syndrome.
Cell research - 1 Oct 2016
Xie Chang, Zhang Ya-Ping, Song Lu, Luo Jie, Qi Wei, Hu Jialu, Lu Danbo, Yang Zhen, Zhang Jian, Xiao Jian, Zhou Bin, Du Jiu-Lin, Jing Naihe, Liu Yong, Wang Yan, Li Bo-Liang, Song Bao-Liang, Yan Yan
Abstract excerpt
PRKAG2 cardiac syndrome is an autosomal dominant inherited disease resulted from mutations in the PRKAG2 gene that encodes γ2 regulatory subunit of AMP-activated protein kinase. Affected patients usually develop ventricular tachyarrhythmia and experience progressive heart failure that is refractory to medical treatment and requires cardiac transplantation. In this study, we identify a H530R mutation in PRKAG2...
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