Erik de Vrieze, theme Sensory Disorders, aims to develop a new form of genetic therapy for patients with hereditary deafness. The therapy is designed for patients with a mutation in DFNA9, the most common mutation found in hereditary deafness in patients in the Netherlands and Belgium.The ZonMw Off Road subsidy is meant to give young researchers the opportunity to test their groundbreaking hypothesis and to develop this innovative idea into a proof of concept. Applications should be characterized by their high-risk profile and unconventional research methods ('high risk, high gain' projects). Specifically, the research should be fundamental (pilot data are not needed), in the field of (bio)medical of health sciences and concerns the development of technologies for a durable health system.
This year ZonMw granted Erik de Vrieze an Off Road subsidy:
Erik de Vrieze, theme Sensory Disorders, aims to develop a new form of genetic therapy for patients with hereditary deafness. The therapy is designed for patients with a mutation in DFNA9, the most common mutation found in hereditary deafness in patients in the Netherlands and Belgium.
We have two copies of most genes: one paternally inherited, the other comes from the mother. Patient with a mutation in DFNA9 have mutation in only one of these copies. De Vrieze: “One normally functional copy of DFNA9 in principle would be sufficient for the generation of functional protein to prevent deafness. However, in this case, the protein translated from the mutated gene sticks to the normal protein rendering the normal protein unable to exert its function. Therefore, patient with only one mutated copy of DFNA9 still develop deafness.
There is currently no therapy for these patients. Erik de Vrieze aims to develop a therapy by making use of CRISPR-Cas technology. With this technique he can specifically block the mutated DFNA9 protein so it no longer sticks to the healthy protein.
Erik de Vrieze
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