Abstract

In vitro modeling of complex neurological disorders

Lawrence Stanton, Associate Director, Genome Institute of Singapore

Finding genes associated with human diseases has benefited greatly from the rapid advancements in genomics and genetics technologies. Two major advances are now greatly impacting our capacity to do functional genomics with human cells: 1) the ability to generate patient-specific, induced pluripotent stem cells (iPSC) and direct their differentiation to disease relevant cell types; and 2) the ability to preform genome editing. These tools are now providing the opportunity to create in vitro models of complex, human diseases, including many neurological disorders, such Parkinson’s Disease (PD), amyotrophic lateral sclerosis (ALS), spinal muscular atrophy (SMA), and spinal bulbar muscular atrophy (SBMA). In this talk, an update will be provided on progress in our lab to model these diseases.