Gene therapy causes leukemia in bubble children

2003/09/01 Elhuyar Zientzia Iturria: Elhuyar aldizkaria

• Medikuntza

Children's disease of bubbles is due to the lack of a gene. In order to find a definitive treatment for the cure of the disease, in 1999 the gene missing ten children of bubbles was introduced, using retrovirus vectors. After 30 months, two of the children have developed leukemia.

According to experts, this is because the virus has hindered the genes that control cell growth.

The severe immunodeficiency that bubble children suffer is the result of a genetic mutation that, if not treated, dies before age twelve. A spinal cord transplant is currently performed to combat this disease, although after transplantation it is necessary to take immunoglobulin throughout life.

A suitable alternative for children without spinal cord for transplantation may be gene therapy. However, for the moment they have not achieved definitive treatment.