Correcting Disease-Causing Genes

Gene-Therapy Successes Spur Hope for Embattled Field


Gene therapy emerged in the 1990s as a potential treatment for immune deficiency diseases by correcting the genes causing the disease. Two studies show that of 16 children who had gene therapy to treat severe combined immune deficiency (SCID), the immune systems of 13 had been restored. However, one of these children developed leukemia as a result of the treatment. The best treatment that is currently being used for SCID is a bone marrow transplant, for which there usually is no matched donor available. Thus, unmatched donors are used, resulting in a success rate of about 70%. The success rate of gene therapy rivals that of bone marrow transplants, according to Bobby Gaspar, a physician from the Institute of Child Health at University College London.

A child in the SCID gene-therapy trial developed leukemia as a result of a part of the vector, or modified virus, used to inject the corrected genes into the boy's cells. 20 children have received gene therapy to treat a form of SCID linked to the X-chromosome, with five developing leukemia and one dying. Gaspar continues to say that gene therapy is a better alternative for X-related SCID treatment in comparison to conventional treatment because 19 of the 20 children treated have survived. Researchers are currently working on creating a vector that does not contain the leukemia-causing component for treating children with X-related SCID as well as Wiskott-Aldrich Syndrome, another immune deficiency.

I found this article to be interesting because it shows advances in the treatment of hereditary diseases. I never would have believed that faulty genes could be corrected prior to reading this article. While it is currently still a risky procedure, it will be interesting to see if researchers can find a better vector to inject the genes. It would also be interesting to see how this treatment can help people with immune deficiencies on a wider scale.