Related Experiment Video
Updated: May 18, 2026

Genetic Profiling and Genome-Scale Dropout Screening to Identify Therapeutic Targets in Mouse Models of Malignant Peripheral Nerve Sheath Tumor
Published on: August 25, 2023
TARGETgene: a tool for identification of potential therapeutic targets in cancer
Chia-Chin Wu1, David D'Argenio, Shahab Asgharzadeh
1Department of Genomic Medicine, The University of Texas MD Anderson Cancer Center, Houston, Texas, United States of America. perwu777@gmail.com
Abstract:
The vast array of in silico resources and data of high throughput profiling currently available in life sciences research offer the possibility of aiding cancer gene and drug discovery process. Here we propose to take advantage of these resources to develop a tool, TARGETgene, for efficiently identifying mutation drivers, possible therapeutic targets, and drug candidates in cancer. The simple graphical user interface enables rapid, intuitive mapping and analysis at the systems level. Users can find, select, and explore identified target genes and compounds of interest (e.g., novel cancer genes and their enriched biological processes), and validate predictions using user-defined benchmark genes (e.g., target genes detected in RNAi screens) and curated cancer genes via TARGETgene. The high-level capabilities of TARGETgene are also demonstrated through two applications in this paper. The predictions in these two applications were then satisfactorily validated by several ways, including known cancer genes, results of RNAi screens, gene function annotations, and target genes of drugs that have been used or in clinical trial in cancer treatments. TARGETgene is freely available from the Biomedical Simulations Resource web site (http://bmsr.usc.edu/Software/TARGET/TARGET.html).
Insights
TARGETgene is a new tool that uses in silico resources to identify cancer drivers, therapeutic targets, and drug candidates. It aids cancer gene and drug discovery through intuitive analysis and validation.
Area of Science:
- Bioinformatics
- Computational Biology
- Genomics
Background:
- In silico resources and high-throughput profiling data are abundant in life sciences.
- These resources can accelerate cancer gene and drug discovery.
Purpose of the Study:
- To develop TARGETgene, a tool for identifying cancer mutation drivers, therapeutic targets, and drug candidates.
- To provide an intuitive platform for analyzing and validating potential cancer targets.
Main Methods:
- Leveraging in silico resources and high-throughput profiling data.
- Developing a tool with a simple graphical user interface for systems-level analysis.
- Enabling users to explore target genes and compounds, and validate predictions using benchmark and curated cancer genes.
Main Results:
- TARGETgene successfully identified potential cancer genes and drug candidates.
- Predictions were validated using known cancer genes, RNAi screens, gene function annotations, and clinical trial drug targets.
- Two applications demonstrated the tool's capabilities.
Conclusions:
- TARGETgene is an effective tool for cancer gene and drug discovery.
- The tool facilitates efficient identification and validation of therapeutic targets.
- TARGETgene is freely available for research use.
More Related Videos
Related Concept Videos
Targeted Cancer Therapies
There are several types of targeted therapies against specific...
Targeted Cancer Therapies
There are several types of targeted therapies against specific...
Pharmacogenomics: Identification of New Drug Targets
Combination Therapies and Personalized Medicine
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...
Drug Discovery: Overview
Transducer Mechanism: Enzyme-Linked Receptors
Major types that are helpful drug targets include:

