Immunodeficiency infants cured by genetic engineering
A new gene therapy method has been used to treat young children born without immunity. The results, published in the New England Journal of Medicine, show significant improvement.Researchers at the University of California, San Francisco, have developed a new therapy to treat a rare genetic disease (Artemis-SCID) in which infants lack immunity and their bodies are unable to fight infections. The first 10 patients with fatal immunodeficiency two years after the transplant "feel excellent," the doctors noted.
The researchers used the children's own stem cells for treatment instead of a traditional bone marrow transplant from a related donor. This approach reduces the amount of chemotherapy that is given before the transplant.
All 10 patients participating in the study were transfused their own stem cells with a corrected Artemis gene. As early as 12 weeks later, all children began to form their own T- and B-cells - the lymphocytes responsible for the immune response. Within a year, four of the children showed complete restoration of T-cell immunity, and two years later, B-cell immunity.
Another five study participants showed significant improvements over alternative therapies. Only one patient required a second bone marrow transplant.
"The course of their disease is already much better than with conventional treatment. I've never seen such results in any of the other children. It's amazing," Mort Cowan, co-author of the study.
Source: Lentiviral Gene Therapy for Artemis-Deficient SCID | NEJM