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Functional-genetic approaches to understanding drug response and resistance

Matthias Hinterndorfer1, Johannes Zuber2

  • 1Research Institute of Molecular Pathology (IMP), Vienna BioCenter (VBC), 1030 Vienna, Austria.

Current Opinion in Genetics & Development
|April 6, 2019
PubMed

Insights

New functional-genetic tools accelerate drug discovery and personalize medicine by enabling rapid target identification and analysis of drug response mechanisms.

Area of Science:

  • Biotechnology
  • Genetics
  • Pharmacology

Background:

  • Drug development is slow and costly.
  • Understanding therapeutic response and resistance is limited.
  • Functional-genetic tools offer new approaches.

Purpose of the Study:

  • Review applications of functional-genetic tools in drug discovery.
  • Discuss their use in analyzing drug response and resistance.
  • Highlight opportunities for personalized medicine.

Main Methods:

  • CRISPR/Cas9
  • RNA interference (RNAi)
  • Targeted protein degradation
  • Time-resolved and single-cell transcriptomics
  • High-throughput screening

Main Results:

  • Functional-genetic tools enable efficient candidate therapeutic target identification.
  • These technologies facilitate evaluation of targets before drug development.
  • They provide insights into response mechanisms and biomarkers for established drugs.

Conclusions:

  • Emerging functional-genetic tools revolutionize drug discovery and development.
  • These methods enhance the understanding of drug response and resistance.
  • They pave the way for personalized therapeutic strategies.

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