A few years ago, a clinical trial began in France in the hope of curing children with a type of genetic immune deficiency called SCID-X1. Children with this disease have a defective gene, called gamma-c, which prevents a subset of the cells of the immune system from forming, and predisposes the children to life-threatening infections. In an attempt to cure the childrenwho would otherwise die at a young agephysicians used gene therapy to provide them with normal gamma-c genes. This particular trial has had striking success as well as tragedy. Eight of the eleven children are currently thriving. However, in two cases the therapy successfully introduced gamma-c genes, but these children have since developed leukemia. In both children, a gamma-c gene inserted next to another gene, called LMO2. The LMO2 gene has previously been linked to leukemia, and scientists speculate that the insertion of the gamma-c gene next to LMO2 may have overstimulated the gene, causing T cells to proliferate in excess. An LMO2 effect, in combination with the proliferation-inducing effects of the gamma-c gene itself, may be the cause of the leukemia in these two patients. Scientists are still investigating other possible causes. From this single trial, it is … Continue reading
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