HMB204H1 Study Guide - Final Guide: Hematopoietic Stem Cell, Drug Test, Whole Genome Sequencing

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Document Summary

Lecture 6 using model organisms to identify novel. Possible reasons of failure to translate chemical screens into approved drugs. Phenotype" based drug screens in model organisms could increase success rate. Increasing cost of drug discovery remain unknown, even after the drug"s activity and efficacy are determined. Recent analysis indicates 62% of first-in-class drugs approved between 1999 and 2008 were discovered using phenotypic assays rather than using target-based assays. Proposed solution to increase number of approved drugs. More emphasis could be put on animal based screening or even on early proof of clinical efficacy in humans, and less on the predictive power of high-affinity binding to the target of a molecule from a static library. Identify disease genes: whole genome sequencing, linkage analysis, generate a disease model by targeting orthologous gene using crispr. Perform chemical screen to suppress phenotype: biotechnology/transgenic to generate fluorescent reporter lines.