Researchers at the University of California, San Francisco, have developed a new therapy method to treat
Researchers used to treatchildren's own stem cells instead of traditional bone marrow transplantation from a related donor. This approach reduces the amount of chemotherapy that is given before transplantation.
All 10 patients in the study received transfuses of their own stem cells with the corrected Artemis gene.After 12 weeks, all babies began to form their own T and B cells, lymphocytes responsible for the immune response.Within a year, four children had been confirmed to have complete restoration of T-cell immunity, and two years later, B-cell immunity.
An additional five study participants experienced significant improvements compared to alternative therapies. Only one patient required a second bone marrow transplant.
The course of their illness is already much better than with conventional treatment. I have never seen such results in any of the other children. This is amazing.
Mort Cowan, study co-author
One of the treated patients. Photo: Barbara Ries, UCSF
All patients participating in the studydiagnosed with one of the types of severe combined immunodeficiency - DCLRE1C/Artemis deficiency. The disease is associated with the absence of the DCLRE1C/Artemis gene. Without this gene, the child's body is unable to repair DNA or produce antibodies.
Traditionally for the treatment of this diseaseused for bone marrow transplantation. But a transplant even from a close relative causes serious complications: rejection of the transplanted cells, the reaction of donor immunity against the child, and others. All of these complications can lead to death after transplantation.
Read more:
First-of-its-kind material withstands 'projectiles' that travel at the speed of sound
A shock wave from the Sun has opened a crack in the Earth's magnetic field
17-year-old engineer came up with a magnetless motor: it can be used in electric vehicles