Discovery and use of retina-specific promoters to rescue photoreceptor function specifically at clinically relevant, advanced stages of disease.
Viral vector-mediated gene silencing of a novel short retinal isoform of Prolactin promotes photoreceptor survival.
PennSorter is a specialized "spike-sorting" software designed to facilitate the precise assignment of electrical signals to cells with enhanced accuracy and efficiency.
AAV-mediated gene therapy approach for the treatment of Leber congenital amaurosis retinopathies resulting from NPHP5 mutations
Recombinant adeno-associated virus (rAAV) expressing the gene for retinitis pigmentosa GTPAse regulation (RPGR) for the functional and structural rescue of photoreceptor cells of the eye as gene therapy for retinal degeneration and vision loss.