Fig. 1.

Schematic flow diagram of AAV vector use for inner ear therapy. A Packaging of plasmids (#1) encoding the gene of interest and a GFP marker requires co-transfection into HEK293T cells with capsid (#2) and helper (#3) plasmids. The capsid plasmid determines AAV particle structure and subtype, and the helper plasmid encodes the adenoviral genes E1, E2, E4, and VA that facilitate viral packaging. B Purified AAV particles are micro-injected into the inner ear of neonatal mice. Imaging and physiological tests evaluate recombinant gene expression, and its functional consequences once the mice reach designated experimental ages