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Improvements in building in vitro pluripotent stem cell-derived disease models

Speaker

Abstract

The discovery of the CRISPR/Cas9 system has transformed research and now allows straightforward alterations in the genome and can be exploited to introduce changes to mimic diseases. This presentation will highlight the use of CRISPR/Cas9 in the generation of disease models in that are relevant for Parkinson’s disease. A review of our ongoing work on the development of pluripotent stem cell (PSC)-based disease models in the neuronal contexts covers which tools can be used to perform CRISPR/Cas9 based genome editing in PSC, how clonal isolation of edited PSC can be dramatically improved, how edited cells are characterized and how differentiation into the relevant cell type is used to reveal cellular phenotypes caused by the genetic changes.


Improvements in building in vitro pluripotent stem cell-derived disease models