Revealing the Molecular Architecture of PWS Through Large Language Models for Targeted Drug Repurposing

Funding Summary

Dr. Singh will apply artificial intelligence to PWS datasets to glean new information about pathways disrupted in PWS and possible targets for therapy.

Dr. Theresa Strong, Director of Research Programs, shares details on this project in this short video clip.

Lay Abstract

Summary of Project Aims and Research Plan
We are embarking on a transformative project to tackle Prader-Willi Syndrome (PWS) through a cutting-edge combination of artificial intelligence (AI) and advanced genomics. Our team aims to create a comprehensive map of how the deletion of certain genes in PWS affects other genes and pathways within cells. By integrating a wealth of data from both general health and PWS-specific sources, we will use powerful computer models to predict critical interactions and identify key points where therapeutic interventions could be effective. Additionally, we plan to scan thousands of existing drugs, as well as millions of potential drugs, to find those that could potentially be applied to treat PWS, significantly speeding up the discovery of treatments.
Importance of the Research
Our research is crucial because it uses innovative methods to address a gap in current treatment options for PWS. The integration of AI with detailed genetic and protein data promises to revolutionize our understanding of PWS at a systems level, identifying new drug targets and accelerating the repurposing of existing drugs. This approach not only enhances our biological understanding but also opens up faster, more cost-effective paths to treatment compared to traditional drug development. It will also enable PWS researchers to efficiently test their hypotheses by identifying the commercially-available molecules most likely to target their protein of interest.
Expected Impact and Next Steps
In the short term (2-5 years), we expect to deliver an integrated map of the PWS subsystem that will incorporate gene expression, protein interactions, and non-coding RNAs. We anticipate identifying multiple potential drug targets and existing drugs that could be redirected to treat PWS. These findings could lead directly to clinical trials and potentially to new, effective treatments soon afterward.
Advancing Therapeutic Development and Clinical Care
This project directly advances therapeutic development by pinpointing precise intervention points within the disease’s protein networks and providing a rapid screening method to identify viable drugs for treatment. By starting with drugs already approved for other conditions, we can quickly move successful candidates into clinical trials, offering new hope for effective treatments; we will then scan much more broadly for other treatment possibilities.