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New Hampshire biologist reacts to gene-editing discovery

By KIMBERLY HOUGHTON
Union Leader Correspondent

August 14. 2017 11:06PM
This sequence of images shows the development of embryos after being injected with a biological kit to edit their DNA, removing a genetic mutation known to cause hypertrophic cardiomyopathy. (Oregon Health & Science University)

Bryan Luikart, an associate professor of molecular and systems biology at Geisel School of Medicine at Dartmouth College.

HANOVER -- A new study reveals that scientists have successfully corrected a dangerous mutation in genes in human embryos — a feat that has the medical field abuzz.

“It is pretty amazing. It is a super-exciting time to be a scientist right now,” said Bryan Luikart, an associate professor of molecular and systems biology at Geisel School of Medicine at Dartmouth College.

The study, which was published in the journal Nature, was detailed in a New York Times report. According to the article, Oregon researchers reported they repaired dozens of human embryos, fixing a mutation that causes a common heart condition that can lead to sudden death later in life.

“The way they have dodged some ethical considerations is that they didn’t go on to have that embryo grow into a person,” said Luikart, explaining that if the embryos with the repaired mutation did have the opportunity to develop, they would be free of the heart condition.

At the Geisel School of Medicine at Dartmouth, Luikart and his colleagues have already been using this concept with mouse embryos, focusing specifically on autism.

Researchers are using the gene-editing method called CRISPR-Cas9 in hopes of trying to more fully understand autism, which he said is the most critical step in eventually finding a cure.

“I think the CRISPR is a tremendous breakthrough. The question really is where and when do you want to use it,” Luikart said. “I have no ethical concerns using it as a tool to better understand biology.”

The new milestone, an example of human genetic engineering, does carry ethical concerns that Luikart said will trigger some debates. He acknowledged that while the advancement of gene-editing technology could eventually stop unwanted hereditary conditions, it also allows for creating babies with smarter, stronger or more attractive traits.

“The ability to do that is now within our grasp more than it has ever been,” he said.

More importantly, the breakthrough could ultimately eliminate diseases, Luikart said. As the technology advances, he said, genetic diseases that are passed down to children may be corrected before the child receives them.

He used another example of a brain tumor, which often returns after it is surgically removed. Now, once the brain tumor is removed, there is the possibility of placing something in the space to edit and fix the mutation that causes the brain tumor in the first place — if physicians are able to find the right cell to edit, Luikart said.

“People are definitely thinking along those lines, or cutting the HIV genome,” said Luikart, who predicts that those advancements will occur in mice within the next decade, and the ability to do that in humans is definitely there.

The big question is whether that can occur without some sort of side effect that was not predicted, he said.

Columbia University Medical Center posted an article earlier this year warning that CRISPR gene editing can cause hundreds of unintended mutations, based on a study published recently in Nature Methods.

This past May, MilliporeSigma announced it has developed a new genome editing tool that makes CRISPR more efficient, flexible and specific, giving researchers more experimental options and faster results that can accelerate drug development and access to new therapies, according to a release.

“CRISPR genome editing technology is advancing treatment options for some of the toughest medical conditions faced today, including chronic illnesses and cancers for which there are limited or no treatment options,” states the release, adding the applications of CRISPR are far ranging — from identifying genes associated with cancer to reversing mutations that cause blindness.

“It is pretty big news,” Luikart said.

khoughton@newstote.com


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