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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.


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Through an IV, Mr Madeux received billions of copies of a corrective gene and genetic tools to cut his DNA in a precise spot. They travel to the liver, where cells use the instructions to make molecular scissors - called zinc finger nucleases - and prepare the corrective gene. The fingers cut the DNA, allowing the new gene to slip in. The new gene then directs the cell to make the enzyme the patient lacked. "We cut your DNA, open it up, insert a gene, stitch it back up. Invisible mending," said Dr Sandy Macrae, president of Sangamo Therapeutics, the Californian company testing this for two metabolic diseases and hemophilia."

Dr David R. Liu, from Harvard University, said the trial marked a new era. “The Hunter syndrome trial, led by a pioneering company, is another reminder that we have entered an era of genome editing," he said. "I’m hopeful that the approach might provide both therapeutic benefits for the patient, as well as valuable insights that inform the development of future editing therapeutics.” But Dr Robin Lovell-Badge, from the Francis Crick Institute, said getting the treatment to work on more complex conditions would be a challenge. "There's a lot of potential for treating liver diseases in this way," he said. "Taking it to more complex things like muscular dystrophy and cystic fibrosis will require a lot more work." The technique has been carried out before but previous procedures have involved cells being taken out of the body to be edited before being returned. However this is impossible to do with cells within organs like the liver, heart or brain.

In this attempt, the genetic therapy has been designed so it becomes active only once it enters Mr Madeux's liver cells. Dr Chester Whitley, one of the doctors working on the trial, told the BBC: "If it works as well as it does in mice, this has huge ramifications. "I'm very optimistic we have a both safe and efficacious way of providing gene therapy." His long-term hope is to perform gene-editing shortly after birth, because an "untreated baby loses 20 IQ points per year".

Mr Madeux's procedure was attempted to test the safety of performing the gene editing before further research is carried into administering the therapy. If he continues to do well a further nine patients will receive the experimental procedure. Dr Helen O'Neill, from UCL's Institute for Women's Health, said: "With any of these trial therapies, patients and their families put a lot of hope into them. This case shows the willingness of the patients to try gene editing as a therapy, which is a positive step." "Zinc finger nucleases are an older and more expensive tool for gene editing than CRISPR genome editing, so success in this particular patient trial would pave the way for this and newer CRISPR based genome editing trials to begin. "I'm very excited, I think it's the first of many patient trials using technologies that could change people's lives"

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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.
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