PCL102H1 Lecture 16: Chemical and Clinical Probes

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Chemical and clinical probes: the only way to be confident that a protein is a suitable target for a therapeutic intervention is after a drug has been developed, (cid:862)hi(cid:374)dsight is al(cid:449)a(cid:455)s 20/20(cid:863, target validation essentially occurs in patients. It is the only public-private partnership worldwide to undertake such a targeted programme of research. Open access chemical probes: characteristics of chemical probes, potent work at low concentration, selective work only on their target, not off-targets, cell permeable cross membranes. Inhibitors of protein function: valuable reagents, used for preclinical target validation. Paradox of chemical probes: more and more, industry depends on academia to discover and validate new targets but the best target validation happens with the best chemical probes -> these chemicals are usually found in industry. I(cid:374)dustr(cid:455) released (cid:862)(cid:271)est(cid:863) (cid:272)o(cid:373)pou(cid:374)ds (cid:455)ears after the(cid:455) ha(cid:448)e (cid:271)ee(cid:374) s(cid:455)(cid:374)thesized a(cid:374)d (cid:272)hara(cid:272)terized; academia often uses non-optimal chemical tools.

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