Gmppb-mutant mice exhibit dystroglycanopathy symptoms that are rescued with GSK3β inhibition or AAV-mediated GMPPB gene replacement

Abstract Mutations in GDP-mannose pyrophosphorylase B (GMPPB) cause dystroglycanopathy, a rare neuromuscular disorder characterized by α-dystroglycan hypoglycosylation, yet the pathogenic mechanisms and therapeutic options remain poorly defined. To dissect the molecular basis of dystroglycanopathy,...

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Bibliografski detalji

Glavni autori:	Ziwei Fu, Tongchao Wang, Chenyang Zhang, Tianyu Qi, Yanyan Chen, Ju Yang, Hua Yang, Bing Yan, Baoming Gong, Weiqiao Lu, Sushan Luo, Ying Liu, Lei Sun, Hao Jiang, Bo Chen, Zhao Zhang, Xiuping Liu, Yuxiang Wang
Format:	Artigo
Jezik:	Inglês
Izdano:	Nature Portfolio 2026-04-01
Serija:	Nature Communications
Online pristup:	https://doi.org/10.1038/s41467-026-71524-7
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