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Relevant Research in the News

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Usher 1F Collaborative is excited to be part of this with testing set to begin in our mouse model of gene therapy developed at the University of Pittsburgh for the vision loss of Usher 1F.

"The research, led by Muna Naash, at the University of Oklahoma Health Sciences Center, with collaborators in Cleveland and Buffalo, discovered a way to deliver known gene therapies directly to the light-sensitive cells affected by this disease. "

Our researchers are working on a cure for Usher 1F using base editing.

"OLX304A is a program to develop a treatment that targets a single gene for RP patients regardless of their disease-causing gene mutation. This strategy is different from the conventional treatment development strategies for RP which target individual disease-causing genes."

"ViGeneron and Biogen this week have announced a global collaboration and licensing agreement in which the partners will develop and commercialize gene therapy products based on adeno-associated virus (AAV) vectors that treat inherited eye disease."

"Investigators from the clinical-stage gene therapy company and the university’s Penn Center for Advanced Retinal and Ocular Therapeutics (CAROT) will collaborate to analyze specific gene therapy targets for glaucoma, optic neuritis, and retinitis pigmentosa, according to a news release."

A Canadian multi-researcher team effort to develop stem cell therapy to restore vision to those with retinitis pigmentosa

"Several diseases are marked by retinal degeneration that eventually leads to blindness, including age-related macular degeneration (AMD) and retinitis pigmentosa. Targeting these diseases with gene and cell therapies is a popular pursuit, though picking the right targets has proven challenging. Researchers at the Centre for Genomic Regulation (CRG) in Barcelona are developing a stem cell treatment for retinal degeneration that focuses on two chemokine receptors, Ccr5 and Cxcr6, which they identified to be important players in cell signaling."

"A group of scientists of the University of California – San Diego (UCSD), in collaboration with colleagues from China, reversed cellular degeneration and restored visual function in two different mouse models of retinitis pigmantosa. To do that, they used gene-editing tool CRISPR/Cas9 to reprogram mutated rod photoreceptors to become functioning cone photoreceptors....The results showed abundant reprogramed cone cells and preserved cellular architecture in the retinas of both mouse models of RP. Additionally, electroretinography testing of rod and cone receptors in live mice show improved function."

Researchers conducted the study for reporting the anatomical and functional outcomes of Argus II retinal prosthesis implantation in Korean patients. Five consecutive end-stage retinitis pigmentosa (RP) patients who underwent Argus II retinal prosthesis implantation and followed for at least 12 months were included. Recipients of the Argus II implant demonstrated profound improvements in functional vision and mobility in addition to good anatomical outcomes and specialized rehabilitation practices.