Dr. David Lillicrap

Dr. David Lillicrap is a professor in the Department of Pathology and Molecular Medicine at Queen’s University. He is the recipient of a Senior Canada Research Chair in Molecular Hemostasis, and a Career Investigator Award from the Heart and Stroke Foundation of Ontario.

Dr. Lillicrap’s research program has focused, for the past 18 years, on the genetics of blood coagulation, and in particular, on the common inherited bleeding disorders, hemophilia and von Willebrand disease. These conditions represent excellent models of monogenic and complex genetic traits, respectively. Dr. Lillicrap’s research program has three major themes: the investigation of various genetic strategies for the treatment of inherited factor VIII deficiency (hemophilia A); the genetic control of von Willebrand factor biosynthesis and the application of molecular genetic information to the clinical management of these inherited bleeding diseases.

Significant progress has been made towards the development of safe and effective approaches of gene therapy for hemophilia A. Dr. Lillicrap’s group has been among the first to show that delivery of therapeutic factor VIII transgenes with two different forms of viral vector can result in long-term phenotypic recovery in the mouse model of the disease, while the "cure" of several hemophilia A dogs in the Queen’s colony is currently the state-of-the-art in this field. Furthermore, recent investigation of ex vivo stem cell-based delivery protocols have also shown early evidence of promise. Finally, the group has also shown that a novel treatment approach utilizing aminoglycoside antibiotics to suppress nonsense mutations in hemophilia has potential.

Dr. Lillicrap’s von Willebrand disease studies are currently focused on a national study of the most common form of this bleeding disease. His group has recently been the first to characterize what appears to be the most frequent recurring mutation resulting in this phenotype. These studies are now expanding to evaluate additional genetic loci contributing to both abnormally high and low levels of von Willebrand factor, important factors in mediating arterial thrombosis and bleeding, respectively.

This research program is internationally recognized for its contributions to the genetics of hemostasis, and the work currently in progress is making important contributions to the development of clinical gene therapy trials for hemophilia and the genetic characterization of von Willebrand disease. These projects are supported by funds from CIHR, the Canada Research Chairs program, the Heart and Stroke Foundation of Ontario, the Stem Cell Network and the Canadian Hemophilia Society. Overall, the program represents an outstanding example of the application of molecular medicine, from precise and elegant demonstration of pathogenetic mechanisms to the development of innovative biological therapies.

For further information, please contact Dr. David Lillicrap using the Email contact form or by phone at 613 548-1304