American scientists from the University of California in Los Angeles were able to treat nine to nine children aged five to nine ages with a serious congenital immune disorder (LAD-I) using experimental genetic treatment. The results of the study have been published scientifically broadcasting New England Journal of Medicine (Nejm).
Lad-ı is a rare genetic disease (one millionth) that leukocytes cannot fight infections due to mutation in the CD18 gene. Without treatment, most children do not live until adulthood. The only affordable method previously – bone marrow transplant – is associated with high risks, including rejection of grafts.
The new therapy uses stem cells of their own patients. They are removed from the blood and modified by using a safe viral vector that provides a corrected version of the CD18 gene. After turning into the body, the cells begin to produce healthy immune bodies.
Thanks to treatment, all patients eliminated chronic infections, skin lesions and inflammation. CD18 protein levels are normalized and the number of leukocytes reached indicators of healthy peers. Therapy did not cause serious side effects.
Six of nine patients were included in 15 years of observations to evaluate long -term effects.
Previously, scientists To create An unexpected way to initiate immunity rejected during blood poisoning.
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Source: Gazeta
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