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
The disease, which manifests in the first few months of life, is often fatal in the first two years. One treatment option is to performa transplant of 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
First, researchers receivedfrom the bone marrow of patients. Then they used a harmless as a vector to bring the cells the correct version of the gene IL2R . Lentiviruses are to . The best known of these is . Then, the cells were returned to patients who also received a small dose of chemotherapy
Gene Therapy Corrects the Immune System
After three to four months, seven of the eight children had normal levels ofT, 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 therapyremoved. The children seemed to grow normally. Some patients had side effects with a low but overall, the technique appeared to be safe. These results appear in the review .
According to thethis 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 . Here four children could stop intravenously; Three of them responded well to vaccinations . Finally, other gene therapies may present a risk of 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 ofand 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