High-throughput tracking enables systematic phenotyping and drug repurposing in C. elegans disease models.

ABSTRACT: There are thousands of Mendelian diseases with more being discovered weekly and the majority have no approved treatments. To address this need, we require scalable approaches that are relatively inexpensive compared to traditional drug development. In the absence of a validated drug target, phenotypic screening in model organisms provides a route for identifying candidate treatments. Success requires a screenable phenotype. However, the right phenotype and assay may not be obvious for pleiotropic neuromuscular disorders. Here, we show that high-throughput imaging and quantitative phenotyping can be conducted systematically on a panel of C. elegans disease model strains. We used CRISPR genome-editing to create 25 worm models of human Mendelian diseases and phenotyped them using a sin

SUBMITTER: O'Brien TJ 

PROVIDER: S-EPMC11709427 | biostudies-literature | 2025 Jan

REPOSITORIES: biostudies-literature