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Development of Orally Bioavailable Therapeutics by the Chloroplast Expression System to Counter Muscle Degeneration, Weakness, and Fibrosis in DMD

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Technical Report,01 Aug 2015,31 Oct 2016

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University of Florida Gainesville United States

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Patients with DMD suffer from progressive muscle weakness and damage, resulting in fibrotic replacement. The goal of this project is to evaluate the therapeutic potential of the anti-fibrotic agents, ACE2Ang 1-7, when produced in plants using a chloroplast expression system. Lyophilized plant material was delivered by oral gavage to the mdx mouse model for DMD. Initial studies were done to ensure that the plant material and protein was orally bioavailable. Further, additional studies confirmed that ACE2 protein accumulated in the circulation over the course of the treatment. Functional assessment of mice treated for 2 weeks showed improved strength in the diaphragm muscles. However, by 2 months of treatment the benefits were reduced back to untreated controls. We are continuing to analyze more outcome measures for these animals in order to understand if the limited benefit is duet to delivery issues, or if the protein itself is only transiently beneficial. Nevertheless, we believe that this delivery strategy may provide a new way to introduce therapeutic proteins for treating neuromuscular disease.

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