Gene Therapy for Stem Cells
December 20, 2011 | Terry Sharrer
In a remarkable clinical experiment, researchers at the UK’s Sanger Institute and the University of Cambridge have demonstrated a successful gene therapy for alpha 1-antitrypsin deficiency using the patient’s own “reprogrammed” skin cells delivered to the liver. Alpha 1-antitrypsin deficiency is implicated in both cirrhotic liver disease and emphysema. This was the first time gene therapy applied to induced pluripotent stem cells has clearly corrected a human disease. MORE