New gene-editing technology successfully cures a genetic blood disorder in mice
Thursday, October 27, 2016 - 09:31
in Health & Medicine
A next-generation gene-editing system 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 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.