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  • A DNA double helix is seen in an undated artist's illustration released by the National Human Genome Research Institute to Reuters on May 15, 2012.

    A DNA double helix is seen in an undated artist's illustration released by the National Human Genome Research Institute to Reuters on May 15, 2012. | Photo: Reuters

Published 15 November 2017

A patient with a genetic defect had their DNA permanently edited through a new and experimental gene therapy technique.

Scientists in California have announced the first attempt to “edit” genes in a patient’s body to cure disease.

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Experts believe that permanently altering a person’s genetic code can eliminate rare and otherwise untreatable diseases.

AP reports that Brian Madeux, a 44-year-old California resident who has a rare disease known as Hunter syndrome, intravenously “received billions of copies of a corrective gene and a genetic tool to cut his DNA in a precise spot.”

People with Hunter syndrome are missing a gene that is responsible for the creation of an enzyme that breaks down certain carbohydrates, meaning that undigested molecules build up inside of cells and damage the body.

“It’s kind of humbling” to be the first to test this, said Madeux. “I’m willing to take that risk. Hopefully, it will help me and other people.”

Results will not be immediately available to researchers and testing is expected to be ongoing for several months.

Scientists have been able to edit patient’s genes in the past but this has been done in a lab scenario where cells are genetically altered and implanted into a patient. However, this marks the first attempt to permanently edit the genes in a patient’s body. If successful, this operation would invigorate the medical frontier of gene therapy.

The tools used in this procedure are called “zinc finger nucleases,” which operates as a tiny scissor that can precisely cut a specific section of a patient’s DNA.

Existing methods of gene therapy are limited - only certain diseases are able to be treated by gene therapy and even then it isn’t a permanent fix. These methods are also imprecise and where a gene is altered is often uncontrollable, resulting in other complications such as an autoimmune response or cancer.

Critics have also warned of the possibility of gene therapy techniques disrupting reproduction, though scientists have stated that this latest technique only allows the new genetic sequence to be latent within the patient’s liver.

Along with the new corrective gene copies, Madeux received a genetic tool to help transplant the corrective gene with precision, something previously unthinkable. This tool has been described as a “mini surgeon.”

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“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 California company testing this for two metabolic diseases and hemophilia. “It becomes part of your DNA and is there for the rest of your life.”

Because of this, this also makes the stakes higher as gene alterations become permanent.

However, experts have argued that the testing phase of this experimental procedure went without a hitch. Dr. Howard Kaufman, a Boston scientist on the National Institutes of Health panel that approved the studies, said that “so far there’s been no evidence that this is going to be dangerous.”

“Now is not the time to get scared,” Kaufman continued.

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