A team of Wisconsin researchers recently demonstrated that bleeding in mice with hemophilia A and inhibitors could be corrected through bone marrow-targeted gene therapy. The lead investigator of the study was Qizhen Shi, MD, PhD, Department of Pediatrics, Medical College of Wisconsin in Milwaukee. She was joined by investigators from the Blood Research Institute, BloodCenter of Wisconsin, and the Children’s Research Institute, Children’s Hospital of Wisconsin. Shi’s research is currently funded through a National Hemophilia Foundation (NHF) grant. She received a 2007-2008 NHF Career Development Award. 

Shi and colleagues removed the bone marrow from mice, genetically modify the stem cells in it, and then reintroduced the marrow to elicit adequate production of factor FVIII (FVIII) in the mice. The FVIII was then delivered into the bloodstream via the marrow-sourced platelets. In lab tests, the majority of the mice with inhibitors showed an improvement in clotting capacity following delivery of the therapy. Although the results were promising, additional animal studies and eventual human trials will need to be compeleted before this therapeutic approach could become a viable option.

The study, “Syngeneic Transplantation of Hematopoietic Stem Cells That are Genetically Modified to Express Factor VIII in Platelets Restores Hemostasis to Hemophilia A Mice with Pre-existing FVIII Immunity,” was published in the October 2008 issue of Blood.

Source: Medical News Today, November 14, 2008