KW-2449 and VPA exert therapeutic effects on human neurons and cerebral organoids derived from MECP2-null hESCs
Abstract 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...
Main Authors: | , , , , , , , |
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Format: | Article |
Language: | English |
Published: |
BMC
2022-12-01
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Series: | Stem Cell Research & Therapy |
Subjects: | |
Online Access: | https://doi.org/10.1186/s13287-022-03216-0 |