Figure 1. High throughput sequencing for drug discovery. As reported in Li et al., a high throughput sequencing platform could aid the discovery of compounds that reverse gene expression changes associated with disease.

In the method, a profile is generated of gene expression changes associated with a disease state. A range of housekeeping- and cytotoxicity-related genes are incorporated into the panel to control for a compound's specificity.

Target cells are loaded into the wells of a microtiter plate, and a different library compound is added to each well [a]. Cells are then lysed, and the resulting lysate is profiled and transferred to a next-generation sequencing system for sequencing and quantification [b]. Compounds are evaluated based on their ability to modulate the expression of disease-associated genes without significantly affecting the expression of housekeeping- and cytotoxicity-related genes [c].

The high throughput nature of the approach allows researchers to screen libraries for compounds that correct disease-linked genetic signatures.