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An <i>in vivo</i> drug repurposing screen and transcriptional analyses reveals the serotonin pathway and GSK3 as major therapeutic targets for NGLY1 deficiency

2021-11-11

Abstract excerpt

NGLY1 deficiency, a rare disease with no effective treatment, is caused by autosomal recessive, loss-of-function mutations in the N-glycanase 1 (NGLY1) gene and is characterized by global developmental delay, hypotonia, alacrima, and seizures. We used an adult Drosophila model of NGLY1 deficiency to conduct an in vivo, unbiased, small molecule, repurposing screen of FDA-approved drugs to identify therapeutic co...

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Literature Corpus work
8f8aaaa6-2ff6-53ba-b84b-4f5544266352
DOI
10.1101/2021.11.10.468087
Open publication

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