Article

A drug repurposing screen reveals dopamine signaling as a candidate therapeutic pathway for PIGA-CDG

2026-04-18

Abstract excerpt

PIGA-CDG is a congenital disorder of glycosylation caused by pathogenic partial loss-of-function variants in the PIGA gene. PIGA encodes an enzyme responsible for the catalytic transfer of N-acetylglucosamine to phosphatidylinositol during the first step of glycosylphosphatidylinositol anchor biosynthesis. Loss of this enzyme has a widespread phenotypic impact, but primarily results in neurological symptoms includ...

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Literature Corpus work: 13b27c10-81f7-5822-9db6-4164a9665a9b
DOI: 10.64898/2026.04.17.719256

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