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Article

A genome-wide RNAi screen identifies potential drug targets in a C. elegans model of α1-antitrypsin deficiency.

Human molecular genetics - 1 Oct 2014

O'Reilly Linda P, Long Olivia S, Cobanoglu Murat C, Benson Joshua A, Luke Cliff J, Miedel Mark T, Hale Pamela, Perlmutter David H, Bahar Ivet, Silverman Gary A, Pak Stephen C

Abstract excerpt

α1-Antitrypsin deficiency (ATD) is a common genetic disorder that can lead to end-stage liver and lung disease. Although liver transplantation remains the only therapy currently available, manipulation of the proteostasis network (PN) by small molecule therapeutics offers great promise. To accelerate the drug-discovery process for this disease, we first developed a semi-automated...

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Identifiers

PMID: 24838285
DOI: 10.1093/hmg/ddu236
PMCID: PMC4159156

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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