Article
In Vivo Base Editing of Scn5a Rescues Type 3 Long QT Syndrome in Mice.
Circulation - 23 Jan 2024
Qi Man, Ma Shuhong, Liu Jingtong, Liu Xujie, Wei Jingjing, Lu Wen-Jing, Zhang Siyao, Chang Yun, Zhang Yongshuai, Zhong Kejia, Yan Yuting, Zhu Min, Song Yabing, Chen Yundai, Hao Guoliang, Wang Jianbin, Wang Li, Lee Andrew S, Chen Xiangbo, Wang Yongming, Lan Feng
Abstract excerpt
BACKGROUND: Pathogenic variants in SCN5A can result in long QT syndrome type 3, a life-threatening genetic disease. Adenine base editors can convert targeted A T base pairs to G C base pairs, offering a promising tool to correct pathogenic variants. METHODS: We generated a long QT syndrome type 3 mouse model by introducing the T1307M pathogenic variant into the Scn5a gene. The adenine base editor was split into 2...
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