Article

Multiplex Epigenome Editing of <i>MECP2</i> to Rescue Rett Syndrome Neurons

2022-12-02

Abstract excerpt

Rett syndrome (RTT) is an X-linked neurodevelopmental disorder caused by loss-of-function heterozygous mutations of Methyl CpG-binding Protein 2 ( MECP2 ) on the X chromosome in girls. Reactivation of the silent wild-type MECP2 allele from the inactive X chromosome (Xi) represents a promising therapeutic opportunity for female patients with RTT. Here, we applied a multiplex epigenome editing approach to reactiva...

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Literature Corpus work: 161ec34f-889d-5077-8528-854d5fbbcbb7
DOI: 10.1101/2022.11.30.518560

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