Click chemistry enables preclinical evaluation of targeted epigenetic therapies
Details
Publication Year 2017-06-15,Volume 356,Issue #6345,Page 1397-1401
Journal Title
Science
Publication Type
Journal Article
Abstract
The success of new therapies hinges on our ability to understand their molecular and cellular mechanisms of action. Here we modify BET bromodomain inhibitors, an epigenetic-based therapy, to create functionally conserved compounds that are amenable to click-chemistry and can be used as molecular probes in vitro and in vivo. Using click-proteomics and click-sequencing we explore the gene regulatory function of bromodomain containing 4 protein (BRD4) and the transcriptional changes induced by BET inhibitors. Studying mouse models of acute leukemia, we use high-resolution microscopy and flow cytometry to highlight the heterogeneity of drug activity within tumor cells located in different tissue compartments. We also demonstrate the differential distribution and effects of BET inhibitors in normal and malignant cells in vivo. This study provides a potential framework for the pre-clinical assessment of a wide range of drugs.
Publisher
AAAS
Research Division(s)
Immunology
PubMed ID
28619718
Terms of Use/Rights Notice
Refer to copyright notice on published article.


Creation Date: 2017-06-26 02:15:32
Last Modified: 2018-07-09 03:36:19
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