gene2drug: a computational tool for pathway-based rational drug repositioning

Area of Science:

• Computational biology
• Pharmacology
• Genomics

Background:

• Drug repositioning accelerates drug discovery by repurposing existing drugs.
• Transcriptional responses to drugs offer molecular insights for computational drug repositioning.

Purpose of the Study:

• To develop a novel computational methodology for rational drug repositioning.
• To prioritize potential drugs based on their impact on target gene pathways.

Main Methods:

• Utilizing transcriptional response data to small molecules.
• Assessing drug impact on gene transcription within relevant pathways.
• In silico validation and comparison with existing methods.
• Experimental validation in primary hyperoxaluria and neurodegenerative disorder models.

Main Results:

• A novel computational method for drug repositioning was developed.
• The method successfully identified potential therapeutic agents.
• Experimental validation confirmed the efficacy in preclinical models for primary hyperoxaluria and neurodegenerative disorders.

Conclusions:

• The developed computational approach offers a rational strategy for drug repositioning.
• This methodology can accelerate the identification of new therapeutic applications for existing drugs.
• The tool is available as a web resource (Gene2drug) and an R package.