Article

Single-dose administration of therapeutic divalent siRNA targeting MECP2 prevents lethality for one year in an MECP2 duplication mouse model

2025-03-29

Abstract excerpt

<h4> Abstract </h4> MECP2 duplication syndrome (MDS) is a rare X-linked neurodevelopmental disorder caused by duplications of the dosage-sensitive methyl-CpG-binding protein 2 (MECP2) gene. Developing effective therapies for MDS is particularly challenging due to the variability in MECP2 expression among patients and the potential risk of inducing Rett syndrome through excessive pharmacological intervention. Red...

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Literature Corpus work: 8996d9c6-8c84-59bf-a7ab-1c3e21c90e60
DOI: 10.1101/2025.03.26.645328

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