Speaker: Dr Matthew Birket:
UKRI Future Leader Fellow (FLF title: ‘Understanding cardiac progenitors to deliver cardiac regenerative medicine and disease modelling’
Title:
Using human pluripotent stem cells to model conditions affecting the heart – a focus on Fabry disease
Abstract:
Human pluripotent stem cells provide a powerful way to understand the mechanisms of heart development and disease. In this talk, I will discuss how our understanding of cardiac cell specification–namely, the differentiation of stem cells into cardiomyocytes or other heart cell types–is accelerating as a result of single cell genomics. Building on these insights, CRISPR–Cas9 library screening is poised to help illuminate the drivers of congenital heart disease.
By employing patient-derived iPSCs and gene-edited isogenic controls in 3D organoid models, we can also investigate diseases that manifest directly within the heart. Based on work I initiated at Sanofi, our lab is using these approaches to study the lysosomal storage disorder Fabry disease, with the aim of identifying cardiac biomarkers and understand its molecular pathophysiology. I will discuss our progress to date and our plans to expand to multi-tissue and multi-modal models.
