GENE THERAPY FOR TREATING USHER SYNDROME

摘要

The present invention proposes a gene therapy approach as a potential curative treatment for the USHER syndrome, in particular for the USH1G syndrome, which is characterized by a profound deafness and a severe vestibular defect in humans. More precisely, the present invention concerns a gene therapy involving administering a vector expressing a SANS protein in a time window that is compatible with human ethics and welfare i.e., in post-natal, infant and adult humans in which the auditory system is completed. The present inventors herein show for the first time that it is possible to restore genetically-impaired auditory and vestibular functions in human beings in subjects suffering from an Usher1G syndrome even when the therapeutic vector is administered at this late stage.