Target Validation

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Target validation involves proving that DNA, RNA or a protein molecule is directly involved in a disease process and can be a suitable target for development of a new therapeutic drug.Pharmaceutical industry experts and analysts agree that drug target validation is the most critical challenge facing large pharmaceutical companies.The key to successful drug target validation in vitro is the ability to knock-down target gene expression effectively and with high specificity.

Target validation technologies A number of technologies including

  • downregulation of gene expression (gene knockdown, antisense, ribozymes and zinc finger proteins)
  • protein inhibition (phage libraries and antibodies)
  • cellular assays
  • chemical genetics
  • combinatorial biology are linked with target validation.

The integration of various technologies is another challenge.