In order to understand the bases of neurologic complications associated with GALT deficiency, we developed an in vitro neuronal model for classic galactosemia. We have generated induced pluripotent stem cells (IiPSC) from individuals with classic galactosemia and differentiated them into neuronal cultures in vitro. We are investigating the molecular and metabolic signature of these cells. By careful examination of these iPSCs and differentiated neurons, we aim to identify the differences in galactosemic neurons compared to control counterparts.
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Earlier Event: July 1313-1030F What Is Metabolomics And How Can It Help Us Understand Classic Galactosemia?
Later Event: July 1313-1030A Parents And Siblings Of Adults With Galactosemia Roundtable