Less Can Be More for Gene Dose and Drug Sensitivity.

Gatz, Susanne A; Shipley, Janet M · Clin Cancer Res · 2015

basic_science · Level V

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Abstract

CDK4 is preclinically validated as a therapeutic target in PAX3-FOXO1 fusion gene-positive rhabdomyosarcomas. Pharmacologic targeting showed sensitivity but, contrary to expectation, CDK4 genomic amplification and overexpression associated with 25% of cases that exhibited the lowest sensitivities. This emphasizes the importance of tumor-specific preclinical studies to define and understand drug sensitivity.

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