MONDAY, April 3 (HealthDay News) -- The use of gene therapy to replace the gp91phox gene found mutated in X-linked chronic granulomatous disease (X-CGD) has led to clinical improvement in two affected adults, according to a report published online April 2 in Nature Medicine.
X-CGD is marked by immunodeficiency due to a defect in the gp91phox gene, which reduces the antimicrobial activity of phagocytes. To determine if gene therapy could be used to correct the mutation, teams led by Manuel Grez, Ph.D., in Frankfurt, Germany, and Reinhard Seger, M.D., Ph.D., in Zurich, Switzerland, collected CD34+ peripheral blood cells from two affected adults, transduced the cells with virus containing wildtype gp91phox, and reinfused them five days later.
The authors found substantial transfer of gp91phox in both individuals that restored oxidase activity in peripheral blood leukocytes and antimicrobial activity in neutrophils. Both individuals had infection prior to the treatment, one fungal and one bacterial, which were nearly resolved 50 days after the treatment.
"Although insertional influences have probably reinforced the therapeutic efficacy in this trial, our results suggest that gene therapy in combination with bone marrow conditioning can be successfully used to treat inherited diseases affecting the myeloid compartment such as CGD," the authors conclude.
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