How the DNA of a third parent can prevent a hereditary disease

How the DNA of a third parent can prevent a hereditary disease

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Mitochondria are, just like in the view, the powerhouses in cells.

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Artur Plawgo/Getty Images/Science Photo Library

Scientists can protect children against being born with certain devastating genetic disorders by creating “three-parents” babies, according to the results of a historical study released on Wednesday.

British researchers used the experimental technology to help families have eight children who seem healthy. They now vary in age of younger than 6 months to older than 2 years.

The families have been plagued by rare but often fatally inherited for generations mitochondrial disordersaccording to two paper Published in The New England Journal of Medicine.

The four boys and four girls, including a set of identical twins, must be followed longer to confirm that the procedure is safe and effective, the researchers say. But the results are praised as a milestone in the search to use advanced genetic technologies to enable more women to have healthy babies.

“Mitochondrial disease can be devastating to the family. It can be tragic,” says Doug TurnbullA professor in neurology that is part of the Newcastle University team that has been developing technology for more than ten years. “This is an important breakthrough – a big step forward.”

The research tension many families, doctors and other scientists.

“I think this is a milestone. It’s groundbreaking work,” says Dietrich heA associate professor of development cell biology at Columbia University that argued for lifting federal limitations on similar research in the US “it’s extraordinary – no doubt about it.”

Worry about risks Temper Hoop

But some critics doubt the approach. They are concerned about risks and whether the approach will open the door to use genetic technologies to manipulate genes in other ways to ever create ‘designer babies’. In addition, such genetic changes can be passed on for generations. So any mistakes can introduce harmful mutations in the human gene pool, they say.

“It’s dangerous,” says Stuart NewmanA professor in cell biology and anatomy at New York Medical College. “It is biologically dangerous. And then it is culturally dangerous because it is the start of biological manipulation that will not only end with the occurrence of certain diseases, but will bloom to a fully -fledged eugenetic program where genes are manipulated to make designer babies.”

The current American regulations would prevent the procedure in this country from being used to produce children. But a doctor from New York reported in 2016 that he had founded a three-parents baby for a Jordanian family in Mexico. Australia has legalized iT. And doctors in some other countries, including Greece And Ukraine have used the technique to try to help infertile women to have babies, although it is unclear that the method for that purpose works.

“I think it will normalize the fact that it is appropriate to take this material and tinker with it, all when pursuing the perfect baby, whatever someone thinks,” says Francois BaylisA leading research professor Emerita at Dalhousie University in Canada.

Defective mobile powerhouses

Mitochondrial disorders can cause serious health problems, including paralysis, heart failure, brain damage, strokes and blindness. Children born with one of these disorders often live short, painful life. The disorders are caused by defects in mitochondrial DNA. The genetic material is the blueprint for structures called Mitochondria, which provide cells of energy. This DNA is only passed on from mother to child.

The solution that is tested by the British scientists is sometimes mentioned “mitochondrial donation. “It is about removing the genes from the core of the fertilized egg of a pair that a healthy baby wants to get. Those genes represent the majority of genetic material in eggs and are responsible for the most properties of a person, such as their appearance. The defective mitochondrial DNA is lagging behind.

The genes of the core of the egg are then injected into a fertilized egg from a healthy woman who has removed all his DNA, except for the healthy mitochondrial DNA of the donor. This is called pronuclear transfer. The resulting embryo can then develop with the healthy donor mitochondria and later implanted in the womb of the woman who wants a healthy, genetically related baby.

As a result, the baby has all the DNA responsible for the most important characteristics of the two parents who try to get a healthy baby, along with a small amount of mitochondrial DNA of the woman who donated the egg. That is why they are sometimes called “three-parents” babies.

Early results are encouraging

In the new study, the babies were born of seven women with a high risk of transferring serious, pathogenic mitochondrial DNA mutations to their descendants. The pathogenic mitochondrial DNA mutations of their mother were either not detectable or present at levels that are probably not sick, the researchers reported.

“A child with one of these disorders can have a lot of pain, suffer all kinds of problems and die. It is really terrible to see your child die slowly at something so bad. It is heartbreaking,” says Robin Lovell-BadgeA development biologist at the Francis Crick Institute in London who has a editorial accompany the papers. “So for women who risk having children with severe mitochondrial diseases, this offers them an option to have children without suffering. It is very encouraging.”

“All children are good and continue to meet their development mile poles,” says Turnbull of Newcastle University.

Baylis, at Dalhousie University, and others are concerned about risks that may not be clear, for the babies themselves, the women who have the babies and the women donating the eggs, such as a dangerous hyperstimulation of their ovaries.

“There are risks for the women who are going to receive the embryo and there are risks for the women who are the donor of the eggs that the Mitochondria will offer, says Baylis.” We don’t know the future. “

She is also worried about attaching so much importance to the needs of couples to only have children with their own genes.

“What you see is this feeling that” my genes are very valuable. My genes are the only ones who are worth reproducing. “And I think that is always worth asking questions,” says Baylis.

Women who run the risk of having children with a mitochondrial condition have other options, including adoption, she argues.

Turnbull acknowledges that the research remains at a relatively early stage, which requires additional follow -up research and monitoring. “The results so far are very encouraging,” he says.

Turnbull argues that reproductive technologies are strongly regulated in Great -Britain and many other countries: “I think there are enough controls and balances in the system to prevent this from becoming a slippery slope for babies to designer babies.”

Others emphasize that the technology distinguishes from gene processing techniques such as CRISPR, which have also evoked fears for designer babies.

“This is completely different,” says Lovell-Badge. “This is the use of a method that avoids a serious disease. If you care about people’s health, the desire of people to have genetically related children, I see no reason why you should not accept these methods.”

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