Base-edited cynomolgus monkeys mimic core symptoms of STXBP1 encephalopathy

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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...

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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9171284/
https://www.ncbi.nlm.nih.gov/pubmed/35283272
http://dx.doi.org/10.1016/j.ymthe.2022.03.001
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