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Article

Genetically Engineered Mouse Models Unveil Mechanisms and Therapeutic Strategies for GMPPB-Associated Dystroglycanopathy

2024-08-16

Abstract excerpt

<title>Abstract</title> <p>The pathogenesis and therapeutic avenues for GDP-mannose pyrophosphorylase B (GMPPB)-associated dystroglycanopathy (DPG) have not been comprehensively characterized. Recent studies have highlighted a significant association between mutations in GMPPB with DGP, a rare disease characterized by neuromuscular phenotypes. To interrogate the molecular mechanisms driving the onset and progress...

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Literature Corpus work
47303842-6eba-5d3d-875c-7abde1ab6b36
DOI
10.21203/rs.3.rs-4502560/v1
Open publication

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Genetically Engineered Mouse Models Unveil Mechanisms and Therapeutic Strategies for GMPPB-Associated DystroglycanopathyDOI 10.21203/rs.3.rs-4502560/v1
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