abstract |
We present here that, in the DFNB9 mouse model (OTOF knock-out mice), cochlear delivery of fragmented cDNA via a dual-AAV vector approach is well administered after the auditory system of these mice has matured (P30) in such mice. We report the first proof-of-principle that it can effectively and long-lastingly correct the overt deaf phenotype of Accordingly, the present invention relates to a full-length autoperlin polypeptide, or functionality thereof, in an inner parental cell, for use in treating a patient suffering from DFNB9 deafness or preventing DFNB9 deafness in a patient having a DFNB9 mutation. A vector system allowing expression of the fragment, wherein the patient is a person with a developed and mature auditory system, eg, a newborn, infant, toddler, teenager or adult. |