Industrializing yeast as a drug repurposing platform for inherited metabolic diseases
Thevandavakkam, M. A.; Long, N. E.; Roel, B. M.; Kantautas, K. A.; Zakin, S.; Duesterberg, V.; Perlstein, E. O.
Show abstract
The development of therapies for rare diseases, particularly inherited metabolic disorders (IMDs), faces significant challenges due to the high cost and lengthy timelines involved. This study presents a yeast-based platform for drug repurposing that capitalizes on the remarkable similarity between yeast and human cellular pathways. This platform enables rapid, cost-effective screening of potential therapeutic compounds for rare diseases, offering a quick turnaround compared to traditional drug development processes. Utilizing a TargetMol library of comprising [~]50% nutraceuticals, our pipeline accelerates translation of promising drug repurposing hits into patient observational studies in as little as 6 months. We demonstrate the efficacy of this platform through three case studies in the context of IMDs, showcasing its potential to uncover novel treatments and reduce the time and expense associated with bringing therapies to patients with rare diseases.
Matching journals
The top 12 journals account for 50% of the predicted probability mass.
Similar papers in this journal
- Identification of a cardiac glycoside exhibiting favorable brain bioavailability and potency for reducing levels of the cellular prion protein 93%
- The Kinase Chemogenomic Set (KCGS): An open science resource for kinase vulnerability identification 92%
- A novel class of allosteric glucosylceramidase beta 1 correctors that reduce cellular stress and enhance lysosomal function 90%
Similar papers in this journal
- A Yeast-Based High-Throughput Screening Platform for the Discovery of Novel pre-mRNA Splicing Modulators 94%
- Small Molecule Channels Harness Membrane Potential to Concentrate Potassium in trk1Δtrk2Δ Yeast 92%
- Photoaffinity ligand of Cystic Fibrosis corrector VX-445 identifies SCCPDH as an off-target 92%
Similar papers in this journal
- Tales of 1,008 Small Molecules: Phenomic Profiling through Live-cell Imaging in a Panel of Reporter Cell Lines 93%
- A Survey of the Kinome Pharmacopeia Reveals Multiple Scaffolds and Targets for the Development of Novel Anthelmintics 92%
- Target deconvolution using in vitro evolution in haploid human cells 91%
Similar papers in this journal
- NaV1.1 and NaV1.6 selective compounds reduce the behavior phenotype in a novel zebrafish model for Dravet Syndrome 92%
- Combining explainable machine learning, demographic and multi-omic data to identify precision medicine strategies for inflammatory bowel disease 91%
- Structure-based drug repositioning explains ibrutinib as VEGFR2 inhibitor 91%
"Similar papers" are the closest papers from that journal in the model's embedding space. They show what the match is built on, but the ranking comes mostly from a classifier over the whole training set, not from these examples alone.