For the First Time, Scientists Used Gene Editing Inside a Living Patient

Henrietta Strickland
November 16, 2017

There also are gene therapies that don't involve editing DNA.

Zinc finger motifWIKIMEDIA, THOMAS SPLETTSTOESSERIn a first, a man has received a therapy aimed at editing the genes inside his body. The therapy aims to permanently change a patient's DNA to combat diseases.

"We cut your DNA, open it up, insert a gene, stitch it back up. Invisible mending", said Dr Sandy Macrae, president of Sangamo Therapeutics, a California-based company testing the therapy for two metabolic diseases and haemophilia. "It becomes part of your DNA and is there for the rest of your life".

The AP reports that on Monday, 44-year-old Brian Madeaux was hooked up to an IV that delivered billions of copies of a corrective gene into his body, along with a genetic tool called zinc finger proteins created to cut his DNA in precisely the right spot to deliver the new genes. As a result, the carbohydrates build up in his body's cells resulting in many health issues such as hernias, bunions, as well as ear, eye and gall bladder problems.

This isn't the first gene-therapy trial, but its method is different from previous attempts. Scientists hope the remade cells will then begin producing the enzyme Madeux now lacks.

The AP reported that only 1 percent of Madeux's liver cells would need to be corrected in order to effectively treat the disease.

If it's successful, it could give a major boost to the fledgling field of gene therapy.

The results of the test will not become clear for three months but already doctors are excited about the potential of a technique that avoids the morally and ethical questions inherent in CRISPR, a gene-editing tool which alters the genetic code of human embryos.

"It's kind of humbling", Madeux tells the Associated Press about being the first to receive such an in vivo gene-editing treatment. "I'm willing to take that risk", he said. "Hopefully it will help me and other people", the publication reported.

Other reports by Click Lancashire

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