PSIDER grant application approved
Recently, the ZonMw-PSIDER grants were awarded that are intended to stimulate stem cell research for rare disorders. DCRT members Ype Elgersma, Annemieke Aartsma-Rus, and Willeke van Roon-Mom are joining forces on the project 'Towards a human iPSC neuronal platform for neurodevelopmental disorder therapeutic discovery.', which is a collaboration between Erasmus MC, Leiden UMC, Utrecht UMC and Nijmegen UMC. The members received 4 million euro.
In this project, patients with a developmental disorder are identified who carry a mutation that is potentially amenable for correction by antisense oligonucleotide (ASO) therapy. In order to develop and test such a personalized medicine, stem cells are generated from the patient's blood cells. These stem cells are then differentiated into brain cells, on which the mutation-specific ASO will be tested to investigate whether the function of the brain cells can be restored.
The researchers hope that this study is the prelude to the routine development of tailor-made treatment for a large group of serious conditions that are hitherto untreatable. In the development of such therapies, DCRT is a very important partner.
published on Jul 7, 2021
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