7 January 2021

Nael Nadif Kasri, Department of Cognitive Neurosciences, Human Genetics theme Neurodevelopmental disorders, received 250,000 Euro as a member of the SCN1A-UP! consortium. The latter is funded by the European Joint Programme on Rare Diseases (EJP-RD), which brings together over 130 institutions from 35 countries and focuses on pre-clinical research to develop effective therapies for rare diseases.

The SCN1A-UP!  project will run for three years, receiving a total of 1.5 million Euro in funding, and aims at developing therapeutic approaches, based on novel gene therapy tools, for SCN1A-related epilepsies.

SCN1A-UP!  involves five academic partners from France (ANR, University of Cote d’Azur), Italy (Institute of Neuroscience, CNR, Italy), Belgium (University of Leuven), Germany (University of Tübingen). Other participants include collaborators (Netherlands, University of Amsterdam) and patient organizations in the Netherlands, France and Italy.

At Radboudumc, the work will focus on the use of induced pluripotent stem cells for screening and testing of novel gene therapies to restore the neuronal properties of patient-derived neurons.

EJP-RD link:  https://www.ejprarediseases.org/index.php/funded-projects-2020/

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