MCW researcher to study gene therapies for hemophilia

A researcher at the Medical College of Wisconsin (MCW) has received a four-year, $1.5 million dollar grant from the National Institutes of Health's National Heart, Lung, and Blood Institute to continue her study of blood platelet gene therapies for hemophilia A, a genetic bleeding disorder.

Qizhen Shi, MD, PhD, associate professor of pediatric hematology at MCW and an investigator at the Blood Research Institute at BloodCenter of Wisconsin, focuses her work on finding therapies for hemophilia A patients, including those with inactivating antibodies. Immune response often precludes any effective therapies to treat the disease.

Dr. Shi's team previously discovered a "clinically translatable gene therapy protocol" - essentially a gene delivery system that introduces the production of therapeutic clotting proteins in platelets by which the inhibition of inactivating antibodies is circumvented.

"The development of inhibitory antibodies against (blood-clotting factors) is not only a severe and important complication of protein replacement therapy, but also a major concern in gene therapy of hemophilia A." Shi said. "Generation of such inhibitors might potentially preclude gene therapy for hemophilia A. In this project, we propose to investigate a novel gene therapy approach that will provide therapeutic FVIII protein and induce immune tolerance for hemophilia A."

Provided by Medical College of Wisconsin