Generation of Tay Sachs cell models and assay approaches for small molecule discovery

National Center For Advancing Translational Sciences

Investigators

Abstract

During this period, four genotyped disease patient fibroblasts have been already reprogramed into iPSC's and fully characterized. The team is moving forward toward differentiation protocols to produce and characterize patient cortical neurons and measure their capacity to accumulate GM2. Once the protocol of differentiation is fully optimized, bulk number of cortical neurons will be produced with the help of a CompacT SelecT™ SC automated stem cell culture system as previous step to optimizing the screening protocol.