In the ‘minigenes’ project, the USH2A gene is artificially made smaller by taking specific parts of the gene and sticking these together (= minigene). This suddenly makes it possible to insert these minigenes into the current vectors for use in genetic therapy.
In this project the therapeutic effect of shortened USH2A protein variants will be tested in the zebrafish model.
The Stichting Ushersyndroom finances this study in 2016. The first results will be published in 2020.
The expertise centre for Usher Syndrome in Radboudumc started in 2019 a natural development study into Usher Syndrome, de CRUSH study.
This is a very important step in the research into a treatment for Usher Syndrome, because this study may substantially shorten the running time for trials. The Stichting Ushersyndroom finances this five-year study with over €257,000,–, made possible by the donations of contributors and co-financing of the Dutch Dr. Vaillantfonds and the Oogfonds.
The Stichting Ushersyndroom finances, with a contribution from CUREUsher from the UK/Ireland and Landelijke Stichting voor Blinden en Slechtzienden (LSBS), the new study into minigenes for USH2C.
The total grant for this study is € 250,000 and this study started in early 2020.