New gene-editing technology successfully cures a genetic blood disorder in mice
Wednesday, October 26, 2016 - 04:01
in Health & Medicine
A next-generation gene-editing system developed by Carnegie Mellon University and Yale University scientists has successfully cured a genetic blood disorder in living mice using a simple IV treatment. Unlike the popular CRISPR gene-editing technique, the new technology can be administered to living animals and it significantly decreases unwanted, off-target gene mutations. The findings, reported in Nature Communications, offer a new therapeutic approach to treat genetic diseases of the blood like beta thalassemia and sickle cell disease by targeting faulty genes in hematopoietic stem cells.