XSCID Gene Therapy for Older Patients

Patients with X-linked severe combined immunodeficiency (XSCID) who do not regain immune system function after bone marrow transplants–particularly from half-matched parent donors–are often faced with no other alternatives. While gene therapy could potentially help, it has thus far only been successful in XSCID infants.

Researchers at the National Institutes of Health have found that older XSCID patients may also benefit from gene therapy.

One patient showed a majority of the corrected gene; the other two showed 3% and 22%. However, all 3 patients reported improvement in their well- being, with reduced symptoms. In the patient with the majority of the corrected gene, there was clear evidence of correction of immune function tests. The treatment has appeared to be safe during the follow up period that is currently approaching 10 to 27 months.

So when will gene therapy be the preferred method of treatment? Tick tock tick tock tick tock.

Medical News Today, June 2, 2006

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