In:
Science Translational Medicine, American Association for the Advancement of Science (AAAS), Vol. 15, No. 679 ( 2023-01-18)
Abstract:
Rett syndrome is a neurodevelopmental disorder that compromises development of motor skills and communication in girls. Qian et al . show that epigenetic editing the methyl CpG-binding protein 2 (MECP2) gene reactivated MECP2 expression from the silenced X chromosome in RTT human embryonic stem cells and derived RTT neurons. This MECP2 reactivation resulted in a functional rescue of RTT neurons in vitro, paving the way for future optimization and testing of the approach in vivo. —CAC
Type of Medium:
Online Resource
ISSN:
1946-6234
,
1946-6242
DOI:
10.1126/scitranslmed.add4666
Language:
English
Publisher:
American Association for the Advancement of Science (AAAS)
Publication Date:
2023
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