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.
The technique could be trialled in humans in as little as one or two years, [Professor Juan Carlos Izpisua Belmonte, who led the work at the Salk Institute in California] predicted, adding that the team were already working on developing therapies for muscular dystrophy.
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