Skeletal Repair and Regeneration via stem cells

Our laboratory is interested in the biology of adult derived stem cells and their potential for skeletal repair and regeneration. The focus of the investigation is to understand engraftment characteristics of the cells and their fate when transplanted in vivo. The research uses animal models of skeletal diseases to explore the potential of stem cell therapy for the skeleton. The current focus is on a brittle bone disease that results from mutations in the genes that encode type I collagen a major structural protein of bone and other connective tissues. Patients with the brittle bone disease (osteogenesis imperfecta) exhibit frequent bone fractures and severe limb deformities). The mouse model of the disease used in the present study exhibits similar clinical features. The adult derived stem cells are isolated from the bone marrow, characterized by examining cell surface markers and differentiation into different cells phenotypes. Besides cell therapy investigations, gene transfer techniques using viral vectors carrying therapeutic genes are employed to deliver genes into the stem cells to investigate possible gene replacement in the patients? stem cells. Stem cells are tracked in vivo using imaging techniques and by examination of the differentiated cell phenotypes. Because most of the brittle bone disease mutations are dominant negative, the laboratory is also interested in inactivation of mutant genes in stem cells using various techniques, for example Ribozymes and SiRNAs.