Synonym of death in the past, HIV is used to cure 'bubble boys' – 04/18/2019 – Balance and Health


American scientists say they used the HIV virus that causes AIDS in a gene therapy that cured eight children with severe combined immunodeficiency (SCID), also known as blister syndrome.

Babies, born with little or no immune protection, now have fully functional immune systems.

The results of the research developed in the USA were published in the scientific journal New England Journal of Medicine.

Children who have this disorder need to live in completely sterile environments to protect themselves from infections – and without treatment, babies often die.

Gene therapy involved collecting stem cells from the baby's bone marrow and correcting the genetic defect in its DNA soon after birth.

Scientists used a modified version of the HIV virus, which was used as a vector to enter cells and change their genetic code by inserting the "corrected" gene.

As the virus is genetically modified, it is not capable of attacking the body.

According to the researchers, most babies were discharged from the hospital a month later.

"These patients are responding to vaccination and have an immune system capable of producing all the immune cells they need to protect themselves against infections while exploring the world and lead normal lives," said study author Ewelina Mamcarz in a statement released by St. Jude Children's Hospital, Tennessee.

"This is unheard of for patients with SCID-X1," he added, referring to the more common type of the disease.

The patients were treated at St. Jude Children's Hospital, Tennessee, and at the UCSF Benioff Children's Hospital in San Francisco.

The case of David Vetter is perhaps the most famous of severe combined immunodeficiency.

Dubbed "Bubble Boy," Vetter was born in 1971 with the disease and died at age 12 after an unsuccessful bone marrow transplant.

Twenty seconds after his birth, he was placed in a plastic isolation chamber to protect himself from infection, where he lived until he was six years old when he was given a special suit developed by NASA, the American space agency.

His parents had already lost a child to the disease before he was born.

Currently, the best treatment for SCID-XI is the bone marrow transplant of a brother or sister who is compatible.

But according to St. Jude Hospital, more than 80% of patients do not have this type of donor and rely on third-party stem cells.

It is less likely in these cases that the procedure will cure the disease – and there is more chance of causing serious side effects as a result of treatment.

Advances in gene therapy offered other alternatives to bone marrow transplantation, but many treatments involved chemotherapy and had implications for a number of other diseases, including blood disorders such as sickle cell anemia, thalassemia, and metabolic syndrome.


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