A paper in the April issue of Nature Medicine reports clinical improvement on two adults who received gene therapy for the treatment of X-linked chronic granulomatous disease (X-CGD).
X-CGD is an immunodeficiency caused by a defect in the oxidative antimicrobial activity of phagocytes -- a subpopulation of cells in the immune system. The disorder results from mutations in the gene gp91phox. The authors found substantial transfer of the gp91phox gene in both individuals, leading to a large number of functionally corrected phagocytes and notable clinical improvement.
Although the sites of the genome in which the gp91phox gene was inserted probably reinforced the therapeutic efficacy in this trial, these results suggest that gene therapy can be useful to treat X-CGD.
Manuel Grez (Georg-Speyer-Haus, Gene Therapy Unit, Frankfurt, Germany)
(C) Nature Medicine press release.
Message posted by: Trevor M. D'Souza