American researchers used the worst HIV virus in the world to heal children. All patients suffered from a gene defect that dishonored them to death in contact with the environment.
Kids born with a combined immunodeficiency – a genetic disorder where the immunity does not work at all and any contact with the outside world could be fatal – were doomed to a sterile life in anticipation of the inevitable, and now they have a carefree childhood. “Without treatment in children born without functioning immune system, very serious infections develop. Most dies in the first year of life. The disease causes a defect in the gene responsible for the functioning of lymphocytes – cells of the immune system”, says the pediatrician-hematologist Evelin Memkarz.
The most famous of these children – David Wetter – was born in the seventies. About the story of a boy, who spent 12 years in a sterile bubble, filmed several films. For a child to leave the hospital outside, NASA researchers even made a special suit for him. David died due to an unsuccessful bone marrow transplant. And the disease was so named – boy’s syndrome in the bubble.
35 years after his death, researchers at the Children’s Hospital of Memphis announced a scientific breakthrough. The human immunodeficiency virus has helped to cure an illness. “We replace the defective gene, take bone marrow cells and insert a normal copy of this gene. The uniqueness of the study is that for the first time, a lentiviral vector is used for the delivery of foreign genes – from a family of viruses that include HIV, “explains researcher Brian Sorrentino.
Still to treat the disease with HIV nobody dared. The methods of gene therapy that were used gave many side effects. Until the publication of sensational results, the researcher did not live for half a year. But how the method works is well known by his colleagues. “He used deactivated HIV. Removed all the active parts and left only the shell to make a healthy copy to the stem cell. It’s much more effective and safer for gene therapy, “explains Evelin Memarkes.
New way of healing already 8 babies. Now they live a normal life and even have an immune response to the vaccine. Such as they are, one is born for 200 thousand and it is exclusively boys. Will they be able to help them all, researchers have not yet predicted, but they say that hope for life beyond the bubble in patients has definitely appeared.