Viral Gene Editing System Corrects Genetic Liver Disease in Newborn Mice
For the first time, researchers have treated an animal model of a genetic disorder using a viral vector to deliver genome-editing components in which the disease- causing mutation has been corrected. Delivery of the vector to newborn mice improved their survival while treatment of adult animals, unexpectedly, made them worse, according to a new study by investigators from the Perelman School of Medicine at the University of Pennsylvania The team published their findings this week in Nature Biotechnology.