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Article

CRISPR activation of the ribosome-associated quality control factor ASCC3 ameliorates fragile X syndrome phenotypes in mice.

Science translational medicine - 8 Oct 2025

Geng Ji, Wang Xiying, Pan Jie, Khan Danish, Pimcharoen Sopida, Zhang Yongjie, Mosammaparast Nima, Hirose Susumu, Petrucelli Leonard, Brandman Onn, Qi Lei S, Lu Bingwei

Abstract excerpt

Loss of fragile X messenger ribonucleoprotein (FMRP) causes fragile X syndrome (FXS), an inherited neurodevelopmental disorder resulting in intellectual disability and autism spectrum disorder; however, the molecular function of FMRP remains uncertain. Here, using cell lines and fibroblasts and induced pluripotent stem cell-derived neurons from healthy individuals and patients with FXS, we showed that FMRP...

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Identifiers

PMID: 41061044
DOI: 10.1126/scitranslmed.adq3551
PMCID: PMC13069991

Genes

ASCC3

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 10 Sept 2026.

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