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Advancing Biological Understanding and Therapeutics Discovery with Small-Molecule Probes.


ABSTRACT: Small-molecule probes can illuminate biological processes and aid in the assessment of emerging therapeutic targets by perturbing biological systems in a manner distinct from other experimental approaches. Despite the tremendous promise of chemical tools for investigating biology and disease, small-molecule probes were unavailable for most targets and pathways as recently as a decade ago. In 2005, the NIH launched the decade-long Molecular Libraries Program with the intent of innovating in and broadening access to small-molecule science. This Perspective describes how novel small-molecule probes identified through the program are enabling the exploration of biological pathways and therapeutic hypotheses not otherwise testable. These experiences illustrate how small-molecule probes can help bridge the chasm between biological research and the development of medicines but also highlight the need to innovate the science of therapeutic discovery.

SUBMITTER: Schreiber SL 

PROVIDER: S-EPMC4564295 | biostudies-literature | 2015 Jun

REPOSITORIES: biostudies-literature

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Advancing Biological Understanding and Therapeutics Discovery with Small-Molecule Probes.

Schreiber Stuart L SL   Kotz Joanne D JD   Li Min M   Aubé Jeffrey J   Austin Christopher P CP   Reed John C JC   Rosen Hugh H   White E Lucile EL   Sklar Larry A LA   Lindsley Craig W CW   Alexander Benjamin R BR   Bittker Joshua A JA   Clemons Paul A PA   de Souza Andrea A   Foley Michael A MA   Palmer Michelle M   Shamji Alykhan F AF   Wawer Mathias J MJ   McManus Owen O   Wu Meng M   Zou Beiyan B   Yu Haibo H   Golden Jennifer E JE   Schoenen Frank J FJ   Simeonov Anton A   Jadhav Ajit A   Jackson Michael R MR   Pinkerton Anthony B AB   Chung Thomas D Y TD   Griffin Patrick R PR   Cravatt Benjamin F BF   Hodder Peter S PS   Roush William R WR   Roberts Edward E   Chung Dong-Hoon DH   Jonsson Colleen B CB   Noah James W JW   Severson William E WE   Ananthan Subramaniam S   Edwards Bruce B   Oprea Tudor I TI   Conn P Jeffrey PJ   Hopkins Corey R CR   Wood Michael R MR   Stauffer Shaun R SR   Emmitte Kyle A KA  

Cell 20150601 6


Small-molecule probes can illuminate biological processes and aid in the assessment of emerging therapeutic targets by perturbing biological systems in a manner distinct from other experimental approaches. Despite the tremendous promise of chemical tools for investigating biology and disease, small-molecule probes were unavailable for most targets and pathways as recently as a decade ago. In 2005, the NIH launched the decade-long Molecular Libraries Program with the intent of innovating in and b  ...[more]

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