Advancing Biological Understanding and Therapeutics Discovery with Small-Molecule Probes

作者：Schreiber Stuart L^*; Kotz Joanne D; Li Min; Aube Jeffrey; Austin Christopher P; Reed John C; Rosen Hugh; White E Lucile; Sklar Larry A; Lindsley Craig W; Alexander Benjamin R; Bittker Joshua A; Clemons Paul A; De Souza Andrea; Foley Michael A; Palmer Michelle; Shamji Alykhan F; Wawer Mathias J; McManus Owen; Wu Meng; Zou Beiyan; Yu Haibo; Golden Jennifer E; Schoenen Frank J; Simeonov Anton; Jadhav Ajit; Jackson Michael R; Pinkerton Anthony B

来源：Cell, 2015, 161(6): 1252-1265.

DOI：10.1016/j.cell.2015.05.023

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.

• 出版日期2015-6-4
• 单位NIH; MIT