Successes and Setbacks of Clinical Gene Therapy

July 30, 2014

(Research in Germany) – The use of retroviral haematopoietic stem cell gene therapy to treat hereditary immunodeficiencies has achieved great success in resolving the actual symptoms of the disease, but many of the patients that underwent therapy developed leukaemia later. Effective gene therapies involving gene shuttles that do not cause cancer are therefore required. The good news is that such therapies already exist.