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Breakthrough as gene-editing technique restores sight to blind animals

(THE GUARDIAN) Hannah Devlin, November 17, 2016 — 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.

Source: Breakthrough as gene-editing technique restores sight to blind animals | Science | The Guardian