A pipeline for drug target identification and validation Conference Paper
| Authors: | Manchado, E.; Huang, C. H.; Tasdemir, N.; Tschaharganeh, D. F.; Wilkinson, J. E.; Lowe, S. W. |
| Title: | A pipeline for drug target identification and validation |
| Conference Title: | 81st Cold Spring Harbor Laboratory Symposium on Quantitative Biology: Targeting Cancer |
| Abstract: | Rapid and affordable tumor profiling has led to an explosion of genomic data that is facilitating the development of new cancer therapies. The potential of therapeutic strategies aimed at inactivating the oncogenic lesions that contribute to the aberrant survival and proliferation of tumor cells has yielded remarkable success in some malignancies such as BRAFmutant melanoma and BCR-ABL expressing chronic myeloid leukemia. However, the direct inhibition of several wellestablished oncoproteins in some of these cancers is not possible or produces only transient benefits. Functional genomics represents a powerful approach for the identification of vulnerabilities linked to specific genetic alterations and has provided substantial insights into cancer signaling networks. Still, as inhibition of gene function can have diverse effects on both tumor and normal tissues, information on the potency of target inhibition on tumor growth as well as the toxic side effects of target inhibition are also needed. Here, we discuss our RNA interference (RNAi) pipeline for cancer target discovery based on our optimized short-hairpin RNA (shRNA) tools for negative selection screens and inducible RNAi platform that, in combination with embryonic stem cell (ESC)-based genetically engineered mouse models (GEMMs), enable deep in vivo target validation. © 2016 Manchado et al. |
| Journal Title | Cold Spring Harbor Symposia on Quantitative Biology |
| Volume: | 81 |
| Conference Dates: | 2016 Jun 1-5 |
| Conference Location: | Cold Spring Harbor, NY |
| ISBN: | 0091-7451 |
| Publisher: | Cold Spring Harbor Laboratory Press |
| Date Published: | 2016-01-01 |
| Start Page: | 257 |
| End Page: | 267 |
| Language: | English |
| DOI: | 10.1101/sqb.2016.81.031096 |
| PROVIDER: | scopus |
| PMCID: | PMC5469697 |
| PUBMED: | 28057848 |
| DOI/URL: | |
| Notes: | Article -- Export Date: 2 August 2017 -- Source: Scopus |
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