Sanford L. Boye

This paper develops and tests dual AAV vectors for treating USH1B by reconstituting full-length MYO7A expression, showing safety and efficacy in non-human primates.

This paper develops a dual-acting RNAi therapy targeting Hif1a and Hif2a to protect retinal cells in AMD models, showing superior efficacy compared to single-target therapies.

The paper develops rAAV capsid mutants with improved tropism for retinal vascular endothelial cells and reports unexpected cardiac transduction in mice.

This paper demonstrates that gene therapy using AAV-mediated NPHP5 augmentation restores photoreceptor structure and function in a canine model of Leber congenital amaurosis.

This study shows that SARM1 depletion prevents NMNAT1-dependent photoreceptor death and retinal degeneration in mice, identifying SARM1 as a therapeutic target for retinopathies.

This study shows that targeting complement C3 with gene therapy reduces neurodegeneration in glaucoma by protecting retinal ganglion cells.