Base editing restores CDKL5 expression and rescues neuronal deficits in a patient-derived model of CDKL5 deficiency disorder

Abstract Cyclin-dependent kinase like 5 (CDKL5) deficiency disorder (CDD) is a rare monogenic neurodevelopmental disorder caused by pathogenic mutations in the CDKL5 gene, with approximately 50% of reported variants being point mutations. Base editing presents a promising therapeutic strategy to cor...

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Detalhes bibliográficos

Principais autores:	Yue Chai, Yao Zhu, Jiayi Zhu, Mingfeng Guan, Zhongyu Zheng, Yu Chen, Hayley Wing Sum Tsang, Tao Ye, Jacque Pak Kan Ip
Formato:	Artigo
Idioma:	Inglês
Publicado em:	Nature Portfolio 2026-04-01
Colecção:	Scientific Reports
Assuntos:	Neurodevelopmental disorder Cyclin-dependent kinase like 5 (CDKL5) CDKL5 deficiency disorder iPSCs Adenine base editing (ABE) Gene correction
Acesso em linha:	https://doi.org/10.1038/s41598-026-48097-y
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