Article

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

2025-04-25

Abstract excerpt

<title>Abstract</title> <p>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 interventio...

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Literature Corpus work: 191a0102-19f7-54c2-859b-016993c36a06
DOI: 10.21203/rs.3.rs-6465542/v1

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