Gene therapy for Leber congenital amaurosis (LCA), an inherited disorder that causes vision loss starting in childhood, improved patients' eyesight and the sensitivity of the retina within weeks of treatment. Both of these ...
May 3, 2015
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Patients who had lost their sight to an inherited retinal disease could see well enough to navigate a maze after being treated with a new gene therapy, according to research presented today at AAO 2017, the 121st Annual Meeting ...
Nov 11, 2017
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A new University of California, Irvine-led study indicates base editing may provide long-lasting retinal protection and prevent vision deterioration in patients with inherited retinal degeneration, specifically in Leber congenital ...
Apr 5, 2022
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Researchers at Case Western Reserve University have developed gene-carrying nanoparticles that home in on target cells and prevent vision loss in mice with a human form of Leber congenital amaurosis.
May 10, 2017
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Researchers from the Massachusetts Eye and Ear Infirmary, The Children's Hospital of Philadelphia, Loyola University Chicago Health Sciences Division and their collaborators have isolated an elusive human gene that causes ...
Jul 29, 2012
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Adults with a genetic form of childhood-onset blindness experienced striking recoveries of night vision within days of receiving an experimental gene therapy, according to researchers at the Scheie Eye Institute in the Perelman ...
Oct 10, 2022
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Scientists at the UCL Institute of Ophthalmology have identified the mechanism behind a common inherited cause of severe sight loss in young children. The results also point to a potential new treatment that may be possible ...
Apr 14, 2016
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University of Wisconsin-Madison engineers have developed methods to observe gene editing in action, and they're putting those capabilities to work to improve genetic engineering techniques.
Jan 24, 2017
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A new study demonstrates success in using a shortened form of the CEP290 gene for gene therapy in a mouse model of Leber congenital amaurosis type 10 (LCA10), a retinal degenerative disorder that causes childhood blindness. ...
Oct 5, 2017
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Independent clinical trials, including one conducted at the Scheie Eye Institute at the Perelman School of Medicine, have reported safety and efficacy for Leber congenital amaurosis (LCA), a congenital form of blindness caused ...
Jan 22, 2013
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