This project is part of the PSIDER Pluripotent Stem cells for Inherited Diseases and Embryonic
Research (PSIDER) project funded by ZonMw. Our Department participates in three PSIDER projects. You will intensively collaborate with the other ethicists in our Department involved in the PSIDER-projects as well as within the researchers in the PSIDER “Human iPSC neuronal platform for neurodevelopmental disorder therapeutic discovery” consortium.
Approximately 1% of children are born with severe neurodevelopmental disorders (NDDs) for which there is currently no disease-modifying therapy. However, because NDDs are frequently caused by identifiable pathogenic mutations, a unique therapeutic opportunity has emerged from recent developments in molecular biology, with the potential to revolutionize the treatment of this group of disorders.
The aim of this PSIDER consortium is the development of anti-sense oligonucleotide (ASO) therapy for neurodevelopmental disorders. Patient-derived hiPSCs will be used to design customized ASOs specific for defined NDD pathogenic mutations to guide selection of lead candidates for advanced secondary (3D brainorganoid) and tertiary (in vivo transplantation) screening as disease modifying treatments in humans.
Embedded in this project is a workpackage responsible for addressing ethical, legal, and societal implications regarding iPSC technology, brain organoids, informed consent (on behalf) of patients with severe NDDs, and first-in-human trials for personalized treatment. You will be tasked to perform the research for this, through both theoretical and empirical research.
You will publish your results in the form of articles and a PhD-thesis. Apart from research tasks, you will be asked to participate in teaching activities and potentially also in ethical advisory boards or committees.
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