We are Laurie Kerkhof and Bianca Zardetto, two new PhD students at the DCRT as part of the PSIDER consortium. The PSIDER consortium focuses on the development of human iPSC neuronal platforms that can be used for therapeutic discovery of neurodevelopmental disorders. In our projects, we will work on rare neurodevelopmental and neurodegenerative disorders and will look for mutations that are potentially amenable for correction by antisense oligonucleotide (ASO) therapy. To facilitate the therapeutic discovery of these rare diseases, stem cells will be generated from patient’s cells and differentiated into brain cells. These brain cells will then be used in a 2D and 3D platform on which the mutation specific ASO can be tested. With the help of these patient-derived cellular disease platforms, we will subsequently investigate whether the function of the brain cells can be restored after ASO delivery.

posted on May 18, 2022