Gene therapy provides temporary relief in patient with immune disorderBMJ 2012; 344 doi: https://doi.org/10.1136/bmj.e1651 (Published 06 March 2012) Cite this as: BMJ 2012;344:e1651
- Susan Mayor
Gene therapy to correct chronic granulomatous disease, a rare genetic condition that impairs immune function, has achieved temporary production of healthy immune cells in a 16 year old boy, enabling him to fight a serious lung infection, Great Ormond Street Hospital for Children has said.
Patients with chronic granulomatous disease have normal protection against viruses but cannot produce functioning phagocytes, which means that they have reduced protection against fungi and bacteria. Affected people are vulnerable to chronic and repeated severe infections and have to take regular medication to reduce their risk and to avoid interaction with other …