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A United States-tested gene therapy for pediatric bladders



In the United States, gene therapy was tested in eight children with severe immunodeficiency. The treatment restored immunity in these young patients, who were unable to benefit from bone marrow transplantation.

Severe combined immunodeficiency associated with X or X-SCID is a rare and serious genetic disease in which immune cells that are designed to fight infections do not develop or function properly. As a result, the patient develops severe respiratory infections. According to the website of Genethon this disease affects one in 200,000 births.

The disease, which manifests in the first few months of life, is often fatal in the first two years. One treatment option is to perform a transplant of marrow from a compatible donor, such as a brother or sister. However, many patients do not have a compatible donor in their environment. For them, gene therapy could therefore be a hope.

This new experimental treatment was developed by scientists at the National Institute of Health of the NIH (1

9459009). To restore young patients' immune system scientists developed gene therapy using a normal copy of the gene IL2R . The eight children of the clinical study aged 2 to 14 months had no siblings who were compatible for a stem cell transplant .

First, researchers received blood stem cells from the bone marrow of patients. Then they used a harmless lentivirus as a vector to bring the cells the correct version of the gene IL2R . Lentiviruses are viruses to RNA . The best known of these is HIV . Then, the cells were returned to patients who also received a small dose of chemotherapy (Busulfan) to implant the modified stem and to produce blood cells. The children were observed for a median duration of 1945 of 16 months.

Gene Therapy Corrects the Immune System

After three to four months, seven of the eight children had normal levels of lymphocytes T, B and NK cells ( natural killer ). In the eighth child, T-lymphocyte levels were initially low, but their numbers increased sharply after a second injection of modified stem cells.

Virus and bacterial infections, which had children before therapy removed. The children seemed to grow normally. Some patients had side effects with a low platelet count but overall, the technique appeared to be safe. These results appear in the review New England Journal of Medicine .

According to the NIH publication this treatment would be safer and more effective than the other gene therapies already tested against this disease using other vectors and other chemotherapies. In particular, these old therapies did not restore all immune cells, such as B cells, to cells that produce antibodies . Here four children IV infusions could stop intravenously; Three of them responded well to vaccinations . Finally, other gene therapies may present a risk of leukemia due to the vector used, which does not appear to be the case with lentivirus.

Therapy Gene May Be an Effective Treatment Option for Infants with This Extremely Severe Disease

For Anthony S. Fauci, Director of the National Institute of NIH of Allergies and Infectious Diseases (Niaid): " These exciting new results suggest that gene therapy may be an effective treatment option for infants with this extremely severe disease, especially for those who do not have an optimal donor for the treatment. "Stem Cell Transplantation." This breakthrough gives them hope To develop a fully functional immune system and lead a healthy and fulfilling life.

What to Remember

  • Eight babies were treated with gene therapy for severe immunodeficiency.
  • Their genetic disorder is related to a lack of an X Chromosome genes together.
  • The gene was transformed from a lentivirus to blood stem cells
  • Lymphocyte scores have improved in patients

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