“Breakthrough as Gene-Editing Technique Restores Sight to Blind Animals” Hannah Devlin, The Guardian, 16 November 2016

“Study first to show gene-editing tool CRISPR can replace faulty genes within adult cells - and in future could be applied to range of devastating genetic diseases.”

Blind animals have had their vision partially restored using a revolutionary DNA editing technique that scientists say could in future be applied to a range of devastating genetic diseases. The study is the first to demonstrate that a gene editing tool, called CRISPR, can be used to replace faulty genes with working versions in the cells of adults - in this case adult rats. Previously, the powerful procedure, in which strands of DNA are snipped out and replaced, had been used only in dividing cells - such as those in an embryo - and scientists had struggled to apply it to non-dividing cells that make up most adult tissue, including the brain, heart, kidneys and liver. The latest advance paves the way for CRISPR to be used to treat a range of incurable illnesses, such as muscular dystrophy, haemophilia and cystic fibrosis, by overwriting aberrant genes with a healthy working version. Professor Juan Carlos Izpisua Belmonte, who led the work at the Salk Institute in California, said: “For the first time, we can enter into cells that do not divide and modify the DNA at will. The possible applications of this discovery are vast.” The technique could be trialled in humans in as little as one or two years, he predicted, adding that the team were already working on developing therapies for muscular dystrophy.The latest study, published in the journal Nature, demonstrates that adult rats that had been engineered to have a genetic form of blindness called retinitis pigmentosa could be treated using CRISPR gene therapy. The condition, which affects about one in 4,000 people, occurs when a faulty gene causes retinal cells to gradually die off, leading to blindness. The scientists targeted the retinal cells by injecting a virus, carrying a package of gene-editing instructions, into the eyes of blind three-week-old rats. “We were able to improve the vision of these blind rats,” said co-lead author Reyna Hernandez-Benitez, also of the Salk Institute. “This early success suggests that this technology is very promising.”

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“Breakthrough as Gene-Editing Technique Restores Sight to Blind Animals” Hannah Devlin, The Guardian, 16 November 2016


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