GENE THERAPY FOR USH1C
Gwenaëlle Géléoc is assistant professor in the Boston Children’s Hospital and the Harvard Medical School. She did research into gene therapy for USH1C. The USH1C gene does the coding for the ‘harmonin’ protein. Géléoc and her colleagues applied the USH1C gene with the help of an AAV vector to mice with mutations in the USH1C gene. Their experiments showed that this type of gene therapy can recover the production of the harmonin protein in the cochlea. The hearing and the balance of these mice almost fully recovered after this treatment. This type of therapy is different from the therapy for USH1C developed by Jennifer Lentz and colleagues.
The therapy of Lentz is only applicable to a specific mutation in the USH1C gene (c.216G>A ), whereas the therapy of Géléoc and colleagues can theoretically be applied to all USH1C patients. Just like Lentz, Géléoc tested her USH1C therapy on mouse models with the c.216G>A mutation in the USH1C gene.