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High-throughput target discovery using cell-based genetics.

P David Jackson1, John J Harrington

  • 1Athersys Inc., 3201 Carnegie Ave., Cleveland, OH 44115, USA.

Drug Discovery Today
|January 29, 2005
PubMed
Summary
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This study proposes a "function-to-gene" approach for high-throughput target discovery. This method prioritizes identifying gene functions through disease models before extensive genomic analysis.

Area of Science:

  • Genomics
  • Molecular Biology
  • Drug Discovery

Background:

  • High-throughput target discovery necessitates effective disease models and genome-wide functional analysis.
  • The post-genomics era has favored "gene-to-function" strategies, leveraging abundant sequence data.
  • Limitations exist in solely relying on "gene-to-function" for comprehensive target identification.

Purpose of the Study:

  • To advocate for a shift towards "function-to-gene" approaches in early-stage target discovery.
  • To emphasize the validation of novel targets through their observed activity in cell-based disease models.
  • To integrate functional insights from disease models with subsequent hypothesis-driven research.

Main Methods:

  • Utilizing cell-based disease models to identify gene functions.

Related Experiment Videos

  • Implementing "function-to-gene" strategies for initial target discovery.
  • Employing hypothesis-driven research for target validation based on functional data.

    • Main Results:

    • The study outlines a framework for prioritizing functional studies in disease models.
    • It suggests that "function-to-gene" can be a more efficient initial step than solely "gene-to-function".
    • The proposed approach aims to accelerate the identification and validation of therapeutic targets.

    Conclusions:

    • A "function-to-gene" approach, followed by hypothesis-driven validation, is recommended for efficient target discovery.
    • Cell-based disease models are crucial for identifying gene functions relevant to disease pathology.
    • This strategy complements existing genomic resources for advancing drug discovery efforts.