Usher 1F Collaborative

David Corey Lab at Harvard Medical School $170,000:-In April, Maryna Invanchenko, MD, PhD, presented at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting. In her presentation she shared the exciting results that the mini gene developed to treat Usher 1F rescued vision in a zebrafish model. -In August, published results of a second strategy, utilizing base editing to successfully restore hearing in an Usher 1F mouse model-In November, published results revealed a third strategy gene therapy employing dual vectors also showed promising results in restoring vision in multiple retinal models, including human retinal organoids and non-human primate retina. -The lab continued its collaboration with the Samuel Pfaff Lab at the Salk Institute for Biological Studies, on a promising gene therapy approach called RNA end-joining (REJ) technology. Monte Westerfield Lab at University of Oregon Institute of Neuroscience $32,500:-In December, published results revealed that a potent antioxidant compound, Hexafluoro, improved visual function and slowed photoreceptor cell death in the Usher 1F zebrafish model. Zubair Ahmed Lab at University of Maryland School of Medicine $25,000:-In December, published results detailed sustained rescue of visual function in a mouse model of Usher 1F utilizing a dual AAV-based gene therapyRUSH1F Natural History Study, $200,000:-44 patients enrolled-Patients from first centers completed year 2 screening with two completing year 3-Patients from later centers added completed year 1 and beginning year 2 In addition, after our initial funding to our research labs, our Harvard team has raised an additional $6.5 million for their Usher 1F research projects, including $250,000 in 2023.