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A drug repurposing screen reveals dopamine signaling as a critical pathway underlying potential therapeutics for the rare disease DPAGT1-CDG

2024-06-12

Abstract excerpt

DPAGT1-CDG is a Congenital Disorder of Glycosylation (CDG) that lacks effective therapies. It is caused by mutations in the gene DPAGT1 which encodes the first enzyme in N-linked glycosylation. We performed a drug repurposing screen on a model of DPAGT1-CDG in Drosophila ("DPAGT1 model") using 1,520 small molecules that are 98% FDA/EMA-approved. We identified 42 candidate drugs that improved the DPAGT1-CDG model....

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Literature Corpus work: c6e761e3-bfff-5d1c-bf6f-3db1f96566d0
DOI: 10.1101/2024.06.12.597770

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