Gene Therapy in Mouse Models of Human Deafness

Here we present a continuation of our successful Foundation Bertarelli project todevelop gene therapy strategies for treatment of genetic hearing loss. In the prior funding period weachieved compelling proof‐of‐concept success demonstrating that inner ear gene therapy can bedeveloped into viable strategies for treating genetic hearing loss. During the next funding period, we willbuild on these advances and extend the project using a multi‐pronged approach that incorporates theexpertise of two physician/scientists and tackles three forms of genetic hearing loss. This expandedcollaboration will accelerate the pace of discovery and translation of this ground‐breaking work into viablestrategies for restoring hearing and balance function in patients who suffer from genetic hearing andbalance disorders.

Specific Aim 1: Optimize gene therapy vectors for gene expression in inner ear hair cells and neurons.
Specific Aim 2: Expand gene therapy trials to include three mouse models of human deafness.
Specific Aim 3: Develop novel strategies for genome editing in the inner ear