To achieve lower and safer vector doses, we are developing novel AAV vectors through AAV capsid engineering to improve transduction efficiency, specificity and evade neutralizing antibodies. In addition, we are now performing an AAV promoter screen for targeting cell types in the inner ear to reduce potential toxicity.

At Otovia, we have established a large-scale method for measurement of CRISPR-Cas activity. Through Site-directed mutagenesis and screening of gene editors, we are now developing  novel gene editing tools, including Cas9 and Cas12, with the advantages of small gene fragments, high activity, and editing range increase. These tools will help us, , to achieve efficient editing in gene therapy for hearing loss.

For the purpose of restoring auditory function in human with hereditary deafness, we use AAV-mediated gene delivery and editing to  repairdefective hair cells, allowing them to function normally.

Inner ear stem cells in culture can mimic the in vivo differentiation of hair cells and supporting cells. Combining AAV, gene editing tools, and stem cell technology, we are searching for important genes affecting hair cell function in high throughput, developing effective methods for functional hair cell regeneration, and formulating drug-making strategies that can be used in humans.