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DE
12 July 2017
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
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.
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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