Dual-AAV delivery of large gene sequences to the inner ear

Adeno-associated viruses (AAVs) are preferred vectors for gene replacement therapy, as they are non-pathogenic, non-inflammatory, induce stable transgene expression in terminally differentiated cells, and a series of natural and engineered capsid proteins can be employed to target the vectors to specific cells. Only one feature of AAVs is limiting: the low cargo capacity for foreign DNA, restricting their application to coding sequences of