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Official PDF TranslationStem Cell Research & Therapy

A novel OTUD5 variant disrupts neural progenitor cell homeostasis: mechanistic insights from HEK293T cell-based analyses

Authors: Na Xu; Shihao Wang; Tingting Yang; Meiping Yu; Yu Sun; Yongkun Zhan; Yongguo Yu

DOI: 10.1186/s13287-026-04974-xStatus: Verified Translated Edition
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Key Findings in This Report

• A novel OTUD5 missense variant (p.Val233Met) was identified in two NDD patients, causing conformational changes in the catalytic OTU domain. • The variant disrupts neural progenitor cell homeostasis by increasing proliferation (1.8-fold) and impairing neuronal differentiation (60% reduction). • Mechanistically, wild-type OTUD5 stabilizes GSK3β by removing K48-linked ubiquitin chains; the mutant shows reduced deubiquitinase activity, accelerating GSK3β degradation. • This study provides a patient-derived iPSC model for testing GSK3β-targeted therapies in OTUD5-related NDDs.