December Experimental Hematology Hemophilia A Manuscript Creates Buzz

A December Experimental Hematology manuscript, Phenotypic correction of hemophilia A in sheep by postnatal intraperitoneal transplantation of FVIII-expressing MSC, has seen much discussion in the literature. Lead author is Christopher D. Porada, Ph.D., associate professor of regenerative medicine at Wake Forest Baptist.

A Wake Forest Baptist news release in early November states: "For the first time, researchers have combined gene therapy and stem cell transplantation to successfully reverse the severe, crippling bleeding disorder hemophilia A in large animals, opening the door to the development of new therapies for human patients. Researchers at Wake Forest Baptist Medical Center's Institute for Regenerative Medicine, collaborating with other institutions, report in Experimental Hematology that a single injection of genetically-modified adult stem cells in two sheep converted the severe disorder to a milder form.”