Article
Base-edited cynomolgus monkeys mimic core symptoms of STXBP1 encephalopathy.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Jun 2022
Lu Zongyang, He Siting, Jiang Jian, Zhuang Ling, Wang Yan, Yang Guang, Jiang Xiaoyu, Nie Yanhong, Fu Jiqiang, Zhang Xiaotong, Lu Yong, Bian Xinyan, Chang Hung-Chun, Xiong Zhiqi, Huang Xingxu, Liu Zhen, Sun Qiang
Abstract excerpt
Presynaptic syntaxin binding protein 1 (STXBP1) is essential for neurotransmitter release. Heterozygous mutations in this protein cause STXBP1 encephalopathy (STXBP1-E), which is characterized by intellectual disabilities and epilepsies. Since nonhuman primates closely resemble humans, monkey models may advance studies on the pathogenesis and therapeutic treatments of STXBP1-E. We generated cynomolgus monkeys...
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