Abstract

Using functional genomics to understand neural degeneration

Lawrence Stanton, Associate Director, Genome Institute of Singapore

Much of the efforts in our lab are geared towards generating patient-specific cells to study the molecular basis of human diseases, primarily neurological disorders. iPSC lines from patients with Parkinson’s disease, amyotrophic lateral sclerosis, spinal muscular atrophy, and spinal bulbar muscular atrophy have been generated or obtained from public sources. A key component of the disease modeling strategy is generating the disease-relevant cell type in vitro. To this end we have optimized directed differentiation methods to turn iPSC into midbrain dopaminergic neurons, motor neurons, and glial cells. These human cells display various phenotypes that one would associate with the disease of origin. Employing functional genomics approaches, we are now characterizing these patient-derived neural cells to elucidate the molecular mechanisms underlying degeneration.