Targeting GNE Myopathy: A Dual Prodrug Approach for the Delivery of N-Acetylmannosamine 6-Phosphate
نویسندگان
چکیده
منابع مشابه
Crystal Structure of the N-Acetylmannosamine Kinase Domain of GNE
BACKGROUND UDP-GlcNAc 2-epimerase/ManNAc 6-kinase, GNE, is a bi-functional enzyme that plays a key role in sialic acid biosynthesis. Mutations of the GNE protein cause sialurea or autosomal recessive inclusion body myopathy/Nonaka myopathy. GNE is the only human protein that contains a kinase domain belonging to the ROK (repressor, ORF, kinase) family. PRINCIPAL FINDINGS We solved the structu...
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ژورنال
عنوان ژورنال: Journal of Medicinal Chemistry
سال: 2019
ISSN: 0022-2623,1520-4804
DOI: 10.1021/acs.jmedchem.9b00833