Speaker:
Title:
Pooled Optical Genetic Screens for Huntington’s Disease Research
Abstract:
Genome wide association studies (GWAS) of Huntington’s disease (HD) age of onset (AOO) have revealed modifiers of HD pathogenesis, but the mechanisms by which they act on HD pathophysiology remain unclear. We are developing a CRISPRi-based platform for measuring the effects of genetic perturbation on these modifiers in induced pluripotent stem cell (iPSC)-derived human neurons. Our approach combines computational methods for choosing targets and optical measurement of tens of proteins and hundreds of cellular phenotypes in a pooled format that can be used to study hundreds of individual gene targets in a single experiment.