Gene therapy for embryos
"In 10 years we will treat hereditary diseases in the womb"
Sergey Kolenov, Hi-tech+
Several teams of scientists have developed a method of treating hereditary diseases in the womb. While we are talking about mice, however, researchers are confident that the technology is also applicable to humans. According to experts, prenatal therapy will become available to people in 10 years.
Within a few weeks, two research groups reported a significant breakthrough in the treatment of hereditary fetal diseases during pregnancy. The team from Yale and Carnegie Mellon University managed to rid mice of the symptoms of beta-thalassemia, a common blood disease.
Their colleagues, whose work Verge tells about, have succeeded in treating another genetic disease, Gaucher syndrome. Among the manifestations of this neurogenerative disorder are joint pain, seizures and cognitive impairment. The disease is caused by mutations in the GBA gene, which plays an important role in the work of lysosomes.
The researchers experimented with a mouse analogue of Gaucher's disease. At the stage of pregnancy, they introduced into mouse embryos a viral vector carrying working copies of the GBA gene. The life span of "edited" mice in the mother's womb increased from 5 to 130 days.
Rodents still have some signs of brain inflammation, so there is no talk of a complete cure. However, this is a promising start.
After the technology is brought to working condition, it can be used for prenatal treatment of a person.
Biologist from New York University's Arthur Kaplan believes that prenatal therapy for hereditary diseases will become available in 10 years.
According to him, it is much easier to treat genetic abnormalities at the embryo stage than to deal with their consequences in adults.
However, the technique has a number of drawbacks. Scientists will have to make sure that it will be safe for the mother. It is not yet known what will happen if the gene editing vectors are embedded in the cells of the uterus. The possibility of immune rejection of the edited fetus should also be excluded. And finally, prenatal editing is clearly not going to be cheap.
The head of Insilico, Alex Zhavoronkov, is skeptical about gene therapy. In his opinion, the way to the treatment of hereditary diseases lies through the use of artificial intelligence. Algorithms will help develop new drugs that can fight the effects of mutations. Consortium will create such neural networks.AI, a joint venture of Insilico and A2A Pharmaceuticals.
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