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Methods Mol Biol. 2013;986:3-13. doi: 10.1007/978-1-62703-311-4_1.

The path to oncology drug target validation: an industry perspective.

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Disease Area Oncology, Novartis Institutes for BioMedical Research, Basel, Switzerland.


The advent of a variety of genomic, proteomic and other system-based scientific approaches has raised the expectations of identifying novel targets for oncology drug discovery. However, the complexity of human genome cancer alterations requires a careful analysis of the function of candidate targets identified by these efforts. The postulation and testing of a hypothesis that modulation of a protein or pathway will result in a therapeutic effect in a preclinical setting is crucial for target validation activities. In this chapter, we provide an overview on target identification and validation approaches to interrogate the functional and therapeutic relevance of a candidate cancer drug target as an essential step towards justifying the subsequent investment in drug discovery efforts.

[Indexed for MEDLINE]

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