Oral Gene Delivery for Hemophilia

This invention is a non-viral trans gene delivery system developed for the long-term treatment of genetic disease. A recombinant plasmid construct with a cDNA of the gene implicated in hemophilia B was used to form nanospheres. Complex coacervation of the recombinant constrct with chitosan, a bioploymer found in the shells of crustaceans, under specific conditions led to the formation of nanospheres. Chitosan is a non-toxic compound used frequently in biomedical applications such as surgical gauze and biodegradable sutures. The chitosan- DNA nanospheres were used for prolonged trans gene expression and protection of the DNA durng gastro-intestinal (GI) delivery. The nanospheres were set in a gelatin matrix to facilitate uptake by ingestion and at a given period after ingested expression of the FIX transgene released from the nanospheres was analyzed in systemic blood and liver tissue. This invention demonstrates that repeated gene delivery through the oral route can compensate for the transient trans gene expression encountered in non-viral delivery. Long-term gene expression is the primary reason for the use of viral vectors in gene therapy, but their use may be no longer be necessar when the gene can be effectively and repeatedly administered in an oral formulation. The use of non-viral vectors in gene therapy is generally considered attactive for safety reasons and this is paricularly important in Hemophilia.

Inventor(s): Leong, Kam W

Type of Offer: Licensing



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