Advances in gene therapy approaches over the past several decades have only recently culminated into approved treatments for a limited number of genetic diseases. The recent approval of a gene therapy for RPE65-associated retinopathy has placed retinal disorders at the forefront of this rapidly advancing translational field. With access to cohorts of well-characterized patients and patient-derived retinal cell models, the Ocular Tissue Engineering Laboratory is ideally positioned to drive new developments in retinal gene therapies. Using the latest available vectors and gene editing technologies, our group is rapidly developing gene therapies for retinal diseases, including Usher syndrome and gene-associated retinopathies.
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