Gene therapy TKID v.2.0

A new gene therapy for severe combined immunodeficiency has been successfully tested

NIH Remedium: Gene Therapy Shows Promise for Severe Combined Immunodeficiency

Researchers from the Dana-Farber Cancer Institute and Boston Children's Hospital have successfully tested a modified gene therapy for X-linked severe combined immunodeficiency (SCID-X1), a form of hereditary immunodeficiency in which patients are extremely vulnerable to infectious diseases and are forced to stay in a sterile environment.

In the New England Journal of Medicine (A Modified gamma-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency) It is reported that 9 children from European and American clinics were selected to participate in the study. All CI participants were injected with CD34+ hematopoietic cells with the SIN-gamma-c viral vector, and their condition was assessed 12 and 38 months after therapy.

According to the results, 8 patients remained alive 38 months after treatment and successfully resisted infections. Only one child died due to an adenovirus infection that developed before his own immune system was restored. In 7 participants, the recovery of the population of peripheral blood T cells was noted, which successfully coped with the pathogens of infections.

According to scientists, this study used a modified viral vector that does not interact with the genes responsible for triggering oncogenesis. In the 90s, attempts were made to genetically treat X-linked severe combined immunodeficiency, but 25% of patients developed leukemia as a result of treatment. It was found that oncogenesis was caused by the peculiarities of the viral vector.

The article emphasizes that the participants of this study will continue to be monitored, since the long-term effect of therapy and its possible impact on the development of leukemia are still unknown.

Portal "Eternal youth" http://vechnayamolodost.ru 10.10.2014