An immune system disorder named X-linked severe combined immunodeficiency, that primarily affects males, is caused by a mutation on the IL2RG gene. Affected people are at high risk for life threatening infections. Blood transplants are the current type of treatment for this immune system disorder, but this does not completely restore their immunity. Using gene therapy, the immunity could be restored much more efficiently. How is this done?
Patient's had stem cells from their bone marrow extracted. From here scientists planted IL2RG genes, that are not affected by the mutation into the stem cells, and they are placed back into the patient. This study was performed on five patients and all displayed improved immune functions. Some better than others though.
This study is in its early stages, so it is not expected fort his technique to be used on the mass. The study was designed to detect safety and effectiveness of the procedure and much more work is needed to be done before any conclusions are met.
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