Dissecting the impact of N-acetylmannosamine (ManNAc) on ganglioside levels in a sialin-deficient cell model

在唾液酸缺乏细胞模型中剖析 N-乙酰甘露糖胺 (ManNAc) 对神经节苷脂水平的影响

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Abstract

Lysosomal free sialic acid storage disorder (FSASD) is an ultra-rare neurodegenerative condition caused by mutations in SLC17A5 , which encodes the lysosomal sialic acid exporter, sialin. Deficiency of sialin leads to lysosomal accumulation of unconjugated ("free") sialic acid. This study investigated the ability of N-acetylmannosamine (ManNAc), a precursor of sialic acid, to rescue glycosphingolipid (GSL) sialylation in a SLC17A5-deficient HEK-293T model system. Our findings reveal that while ManNAc supplementation may enhance sialic acid biosynthesis, it does not fully restore ganglioside sialylation to wild-type levels, highlighting the essential role of lysosomal sialic acid recycling in maintaining GSL sialylation homeostasis.

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