GTO: a comprehensive gene therapy omnibus

Area of Science:

• Biotechnology
• Genomics
• Clinical Research

Background:

• Gene therapy offers a promising treatment approach for genetic disorders.
• Advancements in gene therapy necessitate comprehensive data resources for research acceleration.

Purpose of the Study:

• To develop the Gene Therapy Omnibus (GTO), a centralized database for gene therapy clinical trial and molecular data.
• To facilitate gene therapy research by providing accessible and analyzed datasets.

Main Methods:

• Compiled 6333 clinical trial records and 3466 transcriptome profiles related to gene therapy.
• Included data on 614 altered genes and 22 types of gene therapy (DNA, RNA, cell-based).
• Established 345 comparison datasets, including single-cell RNA-sequencing data, and performed differential gene expression analysis.

Main Results:

• The GTO contains detailed information on gene therapy products, clinical outcomes, and adverse effects.
• Standardized pipelines were used for differential gene expression and functional enrichment analyses.
• A user-friendly interface enables efficient data retrieval, visualization, and analysis.

Conclusions:

• The GTO serves as an invaluable resource for researchers and clinicians in the gene therapy field.
• It supports the investigation of gene therapy mechanisms and clinical applications.
• The comprehensive data and analytical tools in GTO promote further gene therapy innovation.