It’s All in the Delivery: Improving AAV Transfection Efficiency with Exosomes

Abstract

The efficacy of gene therapy treatments for inner-ear disease depends on the specificity and efficiency with which reagents can be introduced into the relevant cells. Several viral vectors, including adenovectors and adeno-associated vectors (AAVs), have been used to shuttle genes into cells of the inner ear,1,2 but relatively low transfection efficiency continues to be an obstacle.3,4 In this issue of Molecular Therapy, Gyorgy et al.5 now show that AAV1 vectors linked to exosomes (exo-AAV1) achieve highly efficient transfection of all types of sensory cells (hair cells) within both the auditory and vestibular sensory epithelia of the inner ear.