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Gene Therapy for Post-Traumatic Osteoarthritis

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Technical Report,30 Sep 2014,29 Sep 2015

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University of Florida Gainsville United States

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We have shown that scAAV vectors have the capacity to deliver exogenous transgenes to the joints of horses with high efficiency, enabling sustained localized expression of therapeutic transgenes at biologically relevant levels for at least 6 months. When delivered into joints with OA symptoms, dramatically higher levels of transgene expression are achieved, particularly in regions of damaged articular cartilage. Based on these preliminary data, we expect this system could be of tremendous benefit in OA, a chronic erosive joint disease, for which there are currently no useful treatments. To provide a clear assessment of the clinical potential of this technology we are testing the following hypothesis scAAV-mediated gene delivery of IL-1Ra to large mammalian joints with chronic, symptomatic OA, will provide sustained long-term therapeutic benefit inhibiting the progression of joint degeneration and improving function and mobility. Additionally, we hypothesize that scAAV. IL-Ra can be delivered to large OA joints with a level of biosafety appropriate for human application. Currently we are half-way through Aim 1 of this proposal and have recruited 1824 horses for the study. Of the recruited horses, 13 have undergone arthroscopic surgery to create an osteochondral defect and 7 of these animals have undergone treatment. Fluids and diagnostics are being collected and analyzed.


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