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Gene therapy treats children with immune disease "bubble"



Ten newborns with rare genetic diseases, known as boy blister disease, were cured with genetic therapy . Treatment seems to completely deprive children of their immune disorder without side effects or complications – the result that scientists have been looking for decades through careful research and disappointing failure.

The previous attempt to use gene therapy for the treatment of severe combined immunodeficiency disease, or SCID, was stopped in 2003 after researchers realized that therapy gave children cancer. The treatment that was released on Wednesday does not have such adverse side effects, and experts hope that it can help cure other rare genetic disorders, such as sickle cell disease.

"This is a change of game," said Jennifer Hemall, a pediatric immunologist. at the Philadelphia Children's Hospital, which was not involved in the study. "For immunologists who follow this disease, gene therapy has always been a hope for the future. It's interesting when this wave of treatment really becomes a reality. "

Infants with SCID are essentially born without functioning of the immune system. Without treatment, they rarely experience their first birthday and can be killed by infections as harmless as a common cold. Such children once remained isolated in sterile conditions, generating the term "boy-bubble". Their unusual situation attracted the attention of national cinematographers and television shows.

Survival in recent years has increased with arrival. large-scale screening tests and refreshing rescue procedures such as bone marrow transplantation. Such treatment has come about with complications, often leaving children dependent on regular infusion of immunoglobulin or causing their new immune system to attack their own body.

New gene therapy – developed by San Francisco's Benioff UCSF Children's Hospital and Children's Hospital and published in the New England Journal of Medicine – corrects the genetic defect of DNA in children after their birth, inducing their bodies to generate missing parts of their immune system.


David Wetter, was born of an inherited disorder of the immune system, shown on this photo on September 11, 1982 in Texas. (AP Photo / ASSOCIATED PRESS)

In an interview, researchers stated that they first extracted blood stem cells from bone marrow infants. They used a modified virus as a means to deliver the correct copy of the defective gene in the stem cells of these patients. Then, these corrected cells were re-poured back into the patient, where they spread and created healthy immune cells.

In order to avoid the accidental inclusion of cancer-causing genes, as was done by previous gene therapy, researchers have incorporated "isolates" into the virus, so that adjacent genes cells will not be affected when the virus enters it in DNA. In another innovation, researchers gave their children a small amount of chemotherapy to clear existing cells from the bone marrow before they were re-embedded in treated cells, giving the adjusted cells more chance of doing.

in the children's research hospital of St. Their team leader and senior author on paper, Brian Sorrentino, spent the last months of their lives in racing against fatal cancer to complete their work on experimental treatments.

Sorrentino was diagnosed at a young age with Hodgkin's lymphoma, saying that his widow, Susanna Sorrentino. Since modern treatments did not exist, his doctors treated him using radiation that weakened his heart and caused lung cancer that killed him in November last year.

"Losing hair, pain and agony of treatment is what made him find new ways to treat other diseases in children," – said his wife in an interview


. Brian Sorrentino holds Gaël José Pino Alva, who was the first a patient who received new gene therapy for SCID. Sorrentino has been working since the 1990s, along with other immunologists, on gene therapy for SCID, but his work has become relevant in the last year and half of life after the diagnosis of cancer, "said Susanna. When he could no longer get to the hospital, his colleagues at St Jude began organizing conference calls and creating a store at Sorrentino's home dining table. At that time, it was obvious that the experimental procedure was successful. Some of their infant patients have developed into infants with a complete normal immune system.

"He told me that he thought he was rescued as a child from Hodgkin, because he thought he should do with his life," said Suzanne, her voice cracked when she mentioned one of her recent conversations with a man "With the litigation, and the children felt so well, he felt that it was a sign that he was reaching what he should.

In recent years, when newborn screening for SCID has become worldwide, experts have found that the disease is more common than previously thought – occurring in one of 50,000

There are several forms of SCID. Gene therapy for the ADA-SCIDS variant is available in Europe. But medications from the most common form – the X-linked SCID that occurs only in boys – has hitherto remained elusive.

The illness attracted national attention in the 1970s in the case of David Weter. News recorded his life when he grew up in a plastic cocoon. His story was born in John Travolta's 1976 film The Boy in a Plastic Bubble. And even after the death of Wetter, something about the infectious effects of the disease kept him in the national consciousness, making the famous episode in the Seinfeld episode and turning into a conceived premise. By this time, the most effective therapy for SCID was the transplantation of donor hematopoietic stem cells that coincides with the tissue, but most patients lack such donors. Donor donors with unidentified donors have also shown that they save lives, but often lead to complications.

The first successful gene therapy trials in 2000 by French physicians gave many researchers and patients hope that they can correct the molecular bases of the disease. just treat his symptoms. But when some of these patients were found leukemia, it temporarily stopped gene therapy trials in America and most of the world and sent heartbreaking researchers to seek answers.

"Leukemia has stopped the field for a long time and rightly so, because people had to know what went wrong," said Jordan Orange, a pediatric immunologist at Columbia University who did not participate in the study of St. Jude , but helped develop gene therapy for another immunodeficiency disease called Wiskott-Aldrich. "But this led us to the point when we finally began to see the actual remedies from incurable conditions."


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