How is therapy that heals 8 "bubble children" with HIV?


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Courtesy of St. Jude Children's Hospital

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Gael was one of the children who benefited from this treatment.

Researchers from the United States say they used the HIV virus to create a gene therapy that cured 8 children suffering from severe combined immunodeficiency, which is commonly known as "blistered children".

The results of the research, developed at the Tennessee hospital in the United States, were published in the journal New England Journal of Medicine.

Babies, who were born with little or no immunity, now have immune systems that function normally.

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Babies who suffer from this condition and do not receive treatment have to live in totally sterile environments and usually die as a child.

In a month

Gene therapy involved removing the bone marrow from infants and correct the genetic defect in your DNA shortly after his birth.

The "correct" gene – used to correct the defect – was inserted into an altered version of the HIV, the virus that causes AIDS.

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Due to his illness, David Vetter lived until he was 6 years old in a plastic chamber. He could only leave it later when NASA designed a special suit to protect it from the environment.

Researchers say most babies are discharged every month.

Ewelina Mamcarz of St. Jude Hospital and co-author of the study said: "These patients are now vaccine-responsive babies and have an immune system to create all the immune cells they need to protect themselves from infections while they explore the world and lead a normal life. "

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"This is a milestone for patients with SCID-X1," he added, referring to the most common type of SCID (severe combined immunodeficiency).

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

What is SCID syndrome?

The case of David Vetter is perhaps the best known of this disease that prevented him from interacting with the outside world outside his plastic chamber.

Baptized as a "bubble boy," Vetter was born in 1971 with this disease and died at age 12 after a failed bone marrow transplant.

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The camera protected the child from contracting any disease, since his body had no defenses

Within 20 seconds after being born at Texas Children's Hospital in Houston, he was placed in a insulation chamber plastic where he lived up to 6 years when he could leave thanks to a plastic suit designed especially for him by NASA.

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His parents had already lost a child because of this disease before he was born.

What other treatment options are there?

Currently, the best treatment is a Bone marrow transplantation from a compatible donor.

But according to St. Jude data, more than 80% of these patients do not receive a compatible donor and must receive stem cells from other donors' blood.

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Vetter died at age 12 after a failed bone marrow transplant.

This process is less likely to cure the disease and is more likely to cause serious side effects as a result of treatment.

Previous advances in gene therapy offered alternatives to bone marrow transplantation, but these treatments sometimes included chemotherapy and had implications for several other diseases, including blood disorders, sickle cell anemia, and thalassemia and metabolic syndrome.

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