Gene therapy is a promising approach for treating many genetic disorders, particularly those such as hemophilia and some metabolic diseases, in which a missing or dysfunctional gene fails to provide a protein required for normal bodily functions. However, the therapeutic potential of gene therapy has been limited by the lack of safe and efficient delivery systems.
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About one in every 5,000 males has hemophilia A, in which a deficiency for a single blood clotting factor leads to severe bleeding after minor injuries and spontaneous bleeding.
Kam Leong, James B. Duke Professor of Biomedical Engineering, is developing a non-viral oral gene delivery technology, a feat that had been considered impossible by many due to the acidic and harsh enzymatic environment presented by the stomach and gastrointestinal tract. The effort is supported by the Duke-Coulter Translational Partners Program and the National Institutes of Health.
Leong's team reported earlier in Nature Medicine that corrective DNA nanoparticles enveloped in chitosan, a natural material derived from the shells of shrimp or other crustaceans, could make it into body tissues and protect mice from an allergic reaction to peanuts. He is now working to optimize the method for the gene-based treatment of hemophilia.