Proposed Treatment To Fix Genetic Diseases Raises Ethics Issues

Posted: October 9, 2013 at 5:44 pm

Will Martin, with his mother, Lori. Will was 2 when Lori learned he was born with Leigh's Syndrome, a progressive genetic condition. So far it's given him low muscle tone and some difficulty with speech.

Mark Sauer of the Columbia University Medical Center, a member of one of two teams of U.S. scientists pursuing the research, calls the effort to prevent infants from getting devastating genetic diseases "noble." Sauer says the groups are hoping "to cure disease and to help women deliver healthy, normal children."

But the research also raises a variety of concerns, including worries it could open the door to creating "designer babies." The Food and Drug Administration has scheduled an Oct. 22 hearing to consider the issues.

Specifically, the research would create an egg with healthy mitochondrial DNA (mtDNA). Unlike the DNA that most people are familiar with the 23 pairs of human chromosomes that program most of our body processes mtDNA is the bit of genetic material inside mitochondria, living structures inside a cell that provide its energy.

Scientists estimate that 1 in every 200 women carries defects in her mtDNA. Between 1 in 2,000 and 1 in 4,000 babies may be born each year with syndromes caused by these genetic glitches; the syndromes range from mild to severe. In many cases, there is no treatment, and the affected child dies early in life.

Lori Martin, 33, of Houston, found out her son, Will, was born with the mitochondrial disease known as Leigh's syndrome, when he was about 2 years old.

"The experience of being told that your son is basically going to die and you don't know when or how, but it's obviously life-changing, and it completely wrecks your world," Martin says.

Martin and her husband are trying to help Will live as long and as happy a life as they can. And they want more kids. But her doctor advised against it.

"Since I am a carrier, I've been told it's recommended I do not have any more biological children using my eggs," Martin says. "It's a really devastating blow to be told not to have more children."

The proposed research might help Martin and women like her.

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Proposed Treatment To Fix Genetic Diseases Raises Ethics Issues

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