A gene-editing first: Scientists try to edit a living human’s DNA

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Scientists for the first time have tried editing a gene inside the body in a bold attempt to permanently change a person’s DNA to cure a disease.

The experiment was done Monday in California on 44-year-old Brian Madeux. Through an IV, he received billions of copies of a corrective gene and a genetic tool to cut his DNA in a precise spot.

Signs of whether it’s working may come in a month; tests will show for sure in three months.

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This time, the gene tinkering is happening in a precise way inside the body. It’s like sending a mini surgeon along to place the new gene in exactly the right location.

Fewer than 10,000 people worldwide have these metabolic diseases, partly because many die very young. Those with Madeux’s condition, Hunter syndrome , lack a gene that makes an enzyme that breaks down certain carbohydrates. These build up in cells and cause havoc throughout the body.

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Weekly IV doses of the missing enzyme can ease some symptoms, but cost $100,000 to $400,000 a year and don’t prevent brain damage.

A gene-editing tool called CRISPR has gotten a lot of recent attention, but this study used a different one called zinc finger nucleases.

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The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion, and analysis. Read full, original post: AP Exclusive: US scientists try 1st gene editing in the body

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