The current PPG is focused on three different preclinical animal models pertinent to developing gene therapy treatment strategies for hemophilia. These animal models are critical towards developing translational strategies for hemophilia gene therapy. The Animal Core will serve as an integral component of the UNC PPG. Activities to support PPG members will include colony breeding, maintenance and genotyping as well as vector construction, administration and characterization in the context of with the ultimate goal of developing clinically relevant gene therapy modalities. Each ofthe mouse strains required by Projects 1 through 4 will be bred and maintained by the Animal Core to facilitate rapid and efficient dissemination to individual projects. Project 3 is focused on evaluating l.A. prevention of hemarthropathy in dogs. All canine model studies will be carried out at the Frances Owen Blood Research Lab and Canine model facility at UNC. Project 4 is focused on developing a non-human primate model of hemophilia B that can be utilized for the study of factor IX deficiency as well as allow for the preclinicaltesting of therapeutic approaches for the treatment of patients with factor IX deficiency. All primate model studies will be carried out at the primate facility at UC Davis.
The current PPG is focused on three different preclinical animal models pertinent to developing gene therapy treatment strategies for hemophilia. These animal models are critical towards developing translational strategies for hemophilia gene therapy. The Animal Core will serve as an integral component of the UNC PPG.
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