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First gene-editing attempted in human body to cure disease

Posted on: 21 November 2017, source: The Telegraph
Doctors have edited a patient's genes in a world-first attempt to alter DNA from within the human body. Medics in California administered the experimental intravenous treatment, which provides the body with instructions to change DNA, on Brian Madeux, who suffers from Hunter's syndrome. People with Hunter's syndrome are unable to break down long sugary molecules called mucopolysaccharides. This leads to the molecules building up and causing brain and organ damage, and in severe cases it can be fatal. Sufferers require regular enzyme replacement therapy to break down the molecules, but Mr Madeux said he opted to take part in a trial as he is "in pain every second of the day". The 44-year-old from Arizona underwent the treatment at UCSF Benioff Children's Hospital in Oakland, California on Monday.