The Efficacy of a Human-Ready mini<i>MECP2</i> Gene Therapy in a Pre-Clinical Model of Rett Syndrome

The Efficacy of a Human-Ready mini<i>MECP2</i> Gene Therapy in a Pre-Clinical Model of Rett Syndrome

Inactivating mutations and the duplication of methyl-CpG binding protein 2 (MeCP2), respectively, mediate Rett syndrome (RTT) and <i>MECP2</i> duplication syndrome. These disorders underscore the conceptual dose-dependent risk posed by <i>MECP2</i> gene therapy for mosaic RTT...

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Bibliographic Details
Main Authors: Chanchal Sadhu, Christopher Lyons, Jiyoung Oh, Indumathy Jagadeeswaran, Steven J. Gray, Sarah E. Sinnett
Format: Article
Language:English
Published: MDPI AG 2023-12-01
Series:Genes
Subjects:
adeno-associated virus (AAV)
methyl-CpG binding protein 2 (MeCP2)
Rett syndrome (RTT)
Online Access:https://www.mdpi.com/2073-4425/15/1/31

Internet

https://www.mdpi.com/2073-4425/15/1/31

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