Gene expression changes in a tumor xenograft by a pyrrole-imidazole polyamide.

Proc Natl Acad Sci U S A, 2012/10/02;109(40):16041-5.

Raskatov JA[1], Nickols NG, Hargrove AE, Marinov GK, Wold B, Dervan PB

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PMID: 22988074DOI: 10.1073/pnas.1214267109

Impact factor: 12.779

Abstract
Gene regulation by DNA binding small molecules could have important therapeutic applications. This study reports the investigation of a DNA-binding pyrrole-imidazole polyamide targeted to bind the DNA sequence 5'-WGGWWW-3' with reference to its potency in a subcutaneous xenograft tumor model. The molecule is capable of trafficking to the tumor site following subcutaneous injection and modulates transcription of select genes in vivo. An FITC-labeled analogue of this polyamide can be detected in tumor-derived cells by confocal microscopy. RNA deep sequencing (RNA-seq) of tumor tissue allowed the identification of further affected genes, a representative panel of which was interrogated by quantitative reverse transcription-PCR and correlated with cell culture expression levels.
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