SINEUP RNA rescues molecular phenotypes associated with CHD8 suppression in autism spectrum disorder model systems.

doi:10.1016/j.ymthe.2024.12.043

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Article

SINEUP RNA rescues molecular phenotypes associated with CHD8 suppression in autism spectrum disorder model systems.

Molecular therapy : the journal of the American Society of Gene Therapy - 5 Mar 2025

Di Leva Francesca, Arnoldi Michele, Santarelli Stefania, Massonot Mathieu, Lemée Marianne Victoria, Bon Carlotta, Pellegrini Miguel, Castellini Maria Elena, Zarantonello Giulia, Messina Andrea, Bozzi Yuri, Bernier Raphael, Zucchelli Silvia, Casarosa Simona, Dassi Erik, Ronzitti Giuseppe, Golzio Christelle, Morandell Jasmin, Gustincich Stefano, Espinoza Stefano, Biagioli Marta

Abstract excerpt

Loss-of-function mutations in the chromodomain helicase DNA-binding 8 (CHD8) gene are strongly associated with autism spectrum disorders (ASDs). Indeed, the reduction of CHD8 causes transcriptional, epigenetic, and cellular phenotypic changes correlated to disease, which can be monitored in assessing new therapeutic approaches. SINEUPs are a functional class of natural and synthetic antisense long non-coding RNAs...

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Identifiers

PMID: 39741407
DOI: 10.1016/j.ymthe.2024.12.043
PMCID: PMC11897779

Genes

CHD8

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