Progress and Promise of Usher Syndrome Society Sponsored Research

Jeffrey Holt, PhD: In 2021 the Usher Syndrome Society (USS) established a Translational Research Grant program. The program was announced with a request for applications and circulated among hearing and vision scientists worldwide. The goal of the grant mechanism is to fund research designed to develop treatments and cures for all forms of Usher syndrome with emphasis on identification of therapeutic strategies and novel therapeutic agents. Particular emphasis has been placed on collaborative efforts that extend across sensory modalities and platform technologies that may advance therapeutic development for multiple forms of USH. In 2023 the second iteration of the USS Translational Research Program was announced. For this presentation, I will summarize progress that has emerged from these USS-sponsored research programs and will outline plans for the coming funding period. The first project, led by Dr. Monte Westerfield aims to screen FDA-approved and experimental drugs for their ability to prevent or slow the death of vision, hearing, and balance cells for all types of Usher syndrome. For the second project, collaborators Drs. Gwenaelle Geleoc and Timothy Yu are developing antisense oligonucleotides to target specific mutations in USH2A.