Source: Recombinetics news release
Recombinetics today announced a collaborative research project with Mayo Clinic to advance Osteogenesis Imperfecta (OI) research by developing the first-ever swine models of OI. These models will help preclinical researchers better understand disease etiology and progression and provide a reliable preclinical model for establishing the safety and efficacy of new therapies that patients with OI desperately need.
Also known as “brittle bone disease”, clinical manifestations of OI vary from a mild increase in fractures to severe bone deformities, hearing loss and death in the neonatal period. To date, no treatment corrects the underlying cause or alleviates the complications of OI.
Currently, research is hampered by mouse models that do not fully recapitulate the disease seen in patients and have been poor predictors of clinical efficacy. Providing the research community with gene-edited swine models that more closely mimic the human disease, could lead to better treatments, including in vivo gene-editing and gene therapy, orthopedic devices for intramedullary rodding, noninvasive techniques for malocclusion, and understanding the natural progression of bone mineralization after currently available therapeutics.
Project efforts will be led by Adrienne Watson, Ph.D., Recombinetics’ Vice President of Research & Development and Mayo Clinic’s David Deyle, MD, partially funded by an SBIR grant from the National Institutes of Health.