Chronic Granulomatous Disease (CGD) is an attractive target for gene therapy since it results from a single gene defect and complete protection does not require full correction of superoxide production, as shown by many healthy X-linked carriers. Gene therapy protocols using lentivectors will start soon in the U.S. and Europe.
A list of current clinical trial is available at Clinical Trials.gov.
Source: Immune Deficiency Foundation Patient & Family Handbook for Primary Immunodeficiency Diseases FIFTH EDITION Copyright 2013 by Immune Deficiency Foundation, USA. This page contains general medical information which cannot be applied safely to any individual case. Medical knowledge and practice can change rapidly. Therefore, this page should not be used as a substitute for professional medical advice