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Relevant Research in the News

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"A new study shows that the complement system, part of the innate immune system, plays a protective role to slow retinal degeneration in a mouse model of retinitis pigmentosa, an inherited eye disease. This surprising discovery contradicts previous studies of other eye diseases suggesting that the complement system worsens retinal degeneration. The research was performed by scientists at the National Eye Institute (NEI), part of the National Institutes of Health, and appears in the Journal of Experimental Medicine."

"Scientists from EPFL in Switzerland and Scuola Superiore Sant'Anna in Italy are developing technology for the blind that bypasses the eyeball entirely and sends messages to the brain. They do this by stimulating the optic nerve with a new type of intraneural electrode called OpticSELINE. Successfully tested in rabbits, they report their results in Nature Biomedical Engineering."

A good summary of the different approaches in research and clinical trial for treating retinitis pigmentosa/Usher Syndrome

Optogenetics is a potential treatment for retinitis pigmentosa that is not dependent on genetic mutation.

"Current strategies for treatment of neurodegenerative disorders are focused on the repair of the primary affected cell type. However, the defective neuron functions within a complex neural circuitry, which also becomes degraded during disease. It is not known whether a rescued neuron and the remodeled circuit will establish communication to regain normal function. We show that the adult mammalian neural retina exhibits a surprising degree of plasticity following rescue of rod photoreceptors. The wayward rod bipolar cell dendrites re-establish contact with rods to support normal synaptic transmission, which is propagated to the retinal ganglion cells. These findings support efforts to repair or replace defective rods in patients blinded by rod cell loss."

"Ophthalmology researchers at the University of Louisville have discovered the loss of vision in RP is the result of a disruption in the flow of nourishing glucose to the rods and cones. This disruption leads to the starvation of the photoreceptors.

"As research provides a better understanding of the progression of RP, this knowledge may lead to therapies that could slow or stop this process before the rods and cones are destroyed."

"A film that generates electric signals in response to light could one day help patients with conditions that cause retinal degeneration, such as retinitis pigmentosa."

"Building on a track record of developing adeno-associated viral (AAV) vectors as a groundbreaking clinical tool for gene therapy and gene editing, researchers report a more sensitive method for capturing the footprint of AAV vectors -- a broad range of sites where the vectors transfer genetic material."

"START therapy is a bold plan to treat inherited blinding eye disease with an eye drop. Here at Fighting Blindness Canada (FBC), we are excited about the research that Dr. Cheryl Gregory-Evans and her team are doing to make START therapy a reality. If successful, START therapy could be used to treat a variety of different genetic eye diseases, including Leber congenital amaurosis (LCA), Usher syndrome, and retinitis pigmentosa."

"The first trial using CRISPR within the human body is to begin this fall to treat a form of blindness, according to the Associated Press. Editas Medicine and Allergan plan to enroll 18 people with Leber congenital amaurosis, a genetic form of blindness that affects the retina, in their study. ...Recruitment to the study is underway at Massachusetts Eye and Ear Infirmary and is to expand to three other sites, according to ClinicalTrials.gov."

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