Application of iPS models of disease to high throughput screening. Cells derived from patients with disease and healthy controls can be used to generate disease-relevant cell types, which can be phenotypically compared with each other. Such cells can be generated in sufficient numbers to be able to perform whole genome genetic screens to identify molecular and cellular mechanisms of disease and therapeutic targets, and also for high throughput drug screening to identify compounds that may be able to revert the disease phenotype. Differences between patient-derived and control cells can be used to identify potential therapeutic targets or agents