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KW-2449 and VPA exert therapeutic effects on human neurons and cerebral organoids derived from MECP2-null hESCs

BACKGROUND: Rett syndrome (RTT), mainly caused by mutations in methyl-CpG binding protein 2 (MECP2), is one of the most prevalent neurodevelopmental disorders in girls. However, the underlying mechanism of MECP2 remains largely unknown and currently there is no effective treatment available for RTT....

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Detalles Bibliográficos
Autores principales: Xu, Ya-Jie, Liu, Pei-Pei, Yan, Zhong-Ze, Mi, Ting-Wei, Wang, Ying-Ying, Li, Qian, Teng, Zhao-Qian, Liu, Chang-Mei
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9795779/
https://www.ncbi.nlm.nih.gov/pubmed/36575558
http://dx.doi.org/10.1186/s13287-022-03216-0