Embryonic Gene Editing Could Wipe Out Several Conditions, Researchers Say

NEW YORK (CBSNewYork) — A major scientific breakthrough was announced this week, and it could wipe out certain diseases.

As CBS2’s Dr. Max Gomez reported, researchers for the first time have fixed a faulty gene in a human embryo. The genetic engineering procedure could be revolutionary, but people are afraid of how it might be used.

Genetic engineering has been around for some years, but using it actually to repair DNA mutations that cause disease is very hard to do.

But now, in a landmark study, researchers from the U.S., China, and Korea have fixed a faulty gene that causes a lethal heart condition.

Medical science has known for many years that certain serious diseases are caused by genetic mutations that are passed along from generation to generation. Researchers have long dreamed of repairing those errors in DNA.

“The goal of preventing and permanently eliminating terrible genetic diseases – we’re talking here cystic fibrosis, Tay-Sachs disease, sickle cell, hemophilia – that’s been a long-sought goal of medicine,” said Dr. Arthur Caplan of NYU Langone Medical Center.

The goal just took a big step toward reality with a study in the journal Nature. It involved using a sperm cell from a male with a mutation known to cause a lethal heart muscle condition, which was used to fertilize an egg without the mutation.

The key was using a new gene-editing technique called Clustered Regularly Interspaced Short Palindromic Repeats – or CRISPR – that found and removed the defective gene.

The unexpected result was that the fertilized egg used the copy of the normal gene from the mothe to repair and replace the edited gene.

And it worked in most of the embryos tested, according to Eric Schadt, dean for precision medicine at the Icahn School of Medicine at Mount Sinai Hospital.

“The efficiency of the overall procedure was much higher than earlier,” Schadt said. “Over 70 percent of the embryos that were targeted were corrected, so it just brought it much closer to prime time and being able to think about its use in clinical applications.”

But like most powerful technologies, the potential for good comes with the possibility for abuse.

“You don’t just fix diseases. You wind up trying to improve or enhance our offspring. You try to make super babies,” Caplan said. “Who’s going to guarantee access? Who’s going to try and guarantee reasonable pricing. There’s a lot of money to be made out here.”

Another caution that both Caplan and Schadt pointed out is that while the embryos looked normal, they were only allowed to develop for a few days in a petri dish.

That is a long way from knowing that an engineered embryo would yield a normal baby. Information about safety is not known.

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