Gene therapy may offer release from sterile isolation for patients lacking immune systems Part 4

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Gene therapy may offer release from sterile isolation for patients lacking immune systems Part 4 (Teacher: Michael)

Because the ADA enzyme is used, for a variety of metabolic processes in the body, it's a more complex disease than other SCIDs. In previous studies, Bordignon , researchers to continue giving patients enzyme replacement therapy for the metabolic problems, even when gene therapy had the immune component of the disorder under control.

The two patients in the Science study never received enzyme replacement therapy, which is not an option for them in their home countries. Bordignon's group observed the build-up of minimal toxic metabolites, indicating that the gene therapy controlling the metabolic side of the disorder as well.

Eventually, Bordignon plans to try using the gene therapy method for treating other diseases, including AIDS.

This study follows earlier research by Marina Cavazzana-Calvo and colleagues, published in April 28, 2000, issue of Science. The French research team used blood stem cells in gene therapy to treat two infants with SCID XI, a variation of the disease caused by a mutation on the X chromosome.

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