In these two Hot Papers, teams led by Mark Kay, a professor of pediatrics and genetics and director of the Human Gene Therapy program at Stanford University, Stanford, Calif.; and pediatrics professor Katherine High, University of Pennsylvania, chose two similar vectors, but different pathways, and achieved comparable results. Kay and his colleagues chose to deliver vector to the liver, while High and her colleagues, following a long tradition, delivered genes intramuscularly.
| Courtesy of Childrens Hospital of Pennsylvania |
 Katherine High |
"If you look at the papers, you see that the maximum levels of clotting factor achieved are the same using either muscle or liver as the target. However, you have to use higher doses [of the intramuscularly injected vector]," says High, who is also director of research, hematology division at Children's Hospital of Philadelphia. That difference could be attributable to differences in the preparation of the vector, she adds.
Kay focused on ...
