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Updated: May 6, 2026

A Non-random Mouse Model for Pharmacological Reactivation of Mecp2 on the Inactive X Chromosome
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A Non-random Mouse Model for Pharmacological Reactivation of Mecp2 on the Inactive X Chromosome

Published on: May 22, 2019

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在脆弱X神经元中持续表观遗传重新激活,使用RNA结合小分子

Christina W Kam1, Jason G Dumelie1, Gabriele Ciceri2,3

  • 1Department of Pharmacology, Weill Medical College, Cornell University, New York, NY 10065, USA.

Genes
|March 28, 2025
PubMed
概括

脆弱X综合征 (FXS) 治疗包括重新激活FMR1基因. 一种新的方法使用短暂的5-aza-dC,其次是2HE-5NMe来维持FMR1表达并逆转FXS症状.

关键词:
是一种RNA结合的小分子.表观遗传学是指表观遗传学.脆弱的X综合征 脆弱的X综合征

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