Research in the news (February/March 2019)
There have been some exciting research developments from around the world in the last few weeks. Here are some stories from our colleagues at other organisations, which we have already …
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New developments in the search for treatments for Stargardt disease
Researchers funded by Retina UK have contributed to the early development of potential new treatments for Stargardt disease, with their findings recently published in the journal Genetics in Medicine. Stargardt …
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Promising interim results for trial of potential new treatment for LCA10
Highly promising interim results from an early phase clinical trial of a potential new treatment for Leber’s congenital amaurosis 10 (LCA10), caused by mutations in the CEP290 gene, have been …
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“First-in-class” treatment for vision loss in Usher syndrome takes a step towards clinical testing
A potentially transformative new treatment for vision loss in Usher syndrome type 2 has been given the go-ahead for initial clinical testing in a small number of adults living with …
First gene editing treatment moves towards clinical trials
The US Food & Drug Administration (FDA) has given the go-ahead for clinical testing of what could be the first gene editing treatment to be used in inherited eye disease. …
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Gene therapy for RPE65 gene approved for use in EU
The gene therapy Luxturna has won European regulatory approval for the treatment of inherited retinal dystrophy caused by mutations in the RPE65 gene. This milestone decision was made by the …
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Ophthalmology clinical research activity on the increase in the UK
Ophthalmology research in the NHS has grown and become more accessible to patients over the last eight years, according to a newly published report in the journal Eye. This is …
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Widespread errors in “proofreading” cause inherited blindness
Mistakes in “proofreading” the genetic code of retinal cells is the cause of a form of inherited blindness, retinitis pigmentosa (RP) caused by mutations in splicing factors. This new understanding …
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First gene therapy for inherited retinal disease takes a step closer to European approval
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended approval of the gene therapy Luxterna for the treatment of inherited retinal dystrophy caused by mutations …
Investigating a revolutionary gene therapy technique for Usher syndrome
Thanks to support from our wonderful community, including those who participated in the Big Give Christmas Challenge last year, we have been able to commit funding to a three-year research …
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Valproic acid no better than placebo for autosomal dominant retinitis pigmentosa
The results of a clinical trial did not provide support for the use of orally administered valproic acid for the treatment of autosomal dominant retinitis pigmentosa, as it did not …
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Scientists Reverse Congenital Blindness in Mice
A team of scientists led by the Icahn School of Medicine at Mount Sinai has successfully reversed congenital blindness in mice by changing supportive cells in the retina called Müller …
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