The invention discloses application of a carrier carrying Pou4f3 genetic information in preparation of a
medicine for treating DFNA15 deafness, and belongs to the technical field of
gene therapy, the DFNA15 deafness is that POU4F3 is mutated, and the POU4F3 is mutated into POU4F3c. 337Cgt; the
medicine for treating the T-type
mutation DFNA15 deafness carries a carrier with Pou4f3 genetic information, the AAV carrier is a combination of AAV-Anc80L65 and a Myo15
promoter, and the AAV carrier is delivered to an
inner ear through
round window membrane injection, targets outer hair cells and compensates for insufficient haplodose of POU4F3. The invention verifies that AAV delivers POU4F3
gene to compensate c.337Cgt for the first time; deafness caused by dominant genetic
mutation of DFNA15 can be corrected due to insufficient haplodose caused by T (p.Q113 *)
mutation, the optimized AAV vector design breaks through the problem of insufficient
hair cell targeting of a traditional vector, the treatment efficiency is remarkably improved, meanwhile, a P60-P180
dynamic monitoring system is established, it is proved that the
curative effect is lasting, no
side effect exists, and a reliable basis is provided for clinical transformation.