A study in the New England Journal of Medicine has reported on the successful health outcomes of 10 children treated by gene therapy for the rare immunodeficiency called Artemis-severe combined immunodeficiency (SCID).
The DNA-repair enzyme Artemis is essential for rearrangement of T- and B-cell receptors and mutations in the gene which encodes Artemis, causes Artemis-SCID and leaves children without a functioning immune system.
Gene therapy has been used before to treat other types of SCID, but the results are significant because the success rates for conventional treatment of this disorder by hematopoietic-cell transplantation (BMT) are poor.
The children in the trial, which took place at the University of California, were all under 5 years old, were given a healthy copy of the Artemis gene into their own bone marrow stem cells. The children involved are now reported to be living at home with their families, attending nursery and preschool, playing outside, and living more normal lives.
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