ZonMw has awarded a PSIDER grant to a consortium led by Steven Kushner and Ype Elgersma from Erasmus MC. The grant is entitled ‘Towards a human iPSC neuronal platform for neurodevelopmental disorder therapeutic discovery’ and is a collaborative project between researchers from Erasmus MC, LUMC, Radboudumc and UMC Utrecht.
Children with a genetic developmental disorder often require lifelong care, which has a major impact on the lives of patients, loved ones and society. In this project researchers will try to find a solution by repairing the genetic mutation using so-called ‘antisense oligonucleotides’.
The goal is to develop a platform for identifying a successful treatment for developmental disorders by comparing induced pluripotent stem cells (iPSCs) from patients with controls. The iPSCs are differentiated to human brain cells with the same genetic make-up as the patient, made from blood cells of that same patient. Also the ethical issues related to the technology will be explored.
Ultimate aim is to deploy the platform to achieve successful therapy.
Ype Elgersma (left) and Steven Kushner (right)
For more information see the ZonMw website.