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Improved Expression of Factor IX in Gene Therapy Vectors

详细技术说明

A portion of this had been licensed:Improving expression levels of human Factor IX is a key for making gene therapy of hemophilia B a reality. This technology provides two mechanisms for improving

*Abstract

A portion of this had been licensed:Improving expression levels of human Factor IX is a key for making gene therapy of hemophilia B a reality. This technology provides two mechanisms for improving Factor IX expression. The first is the use of a specific Factor IX polynucleotide coding sequence designed for optimal expression in human cells. The second is the use of transcriptional regulatory regions minimized in size so that they can be used to express Factor IX, as well as any other gene of interest, in a size-constrained environment such as in a self complementary gene therapy vector system.However this is still available and we hope to commercialize it:Description A liver specific promoter with regulatory regions minimized in size is provided, that can be used to improve expression levels - a key for making gene therapy work in humans. This technology is already being used to improve expression levels of human Factor IX to making gene therapy of hemophilia B a reality; and we believe it can be further used to express any gene of interest in a size-constrained environment, such as in a self-complementary gene therapy vector system. Related scientific references: Nathwani, A.C. et al., "Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver", Blood 107(7): 2653-61 (April 1, 2006).Granted Patents or Published Applications US Patent No. 8,030,065; 8,168,425; Multiple foreign rights. Licensing Opportunities This promoter has been licensed for use in gene therapy vectors to treat hemophilia B. We are currently seeking licensing opportunities for all other uses, with a specific interested in the use of this promoter in gene therapy vectors to drive liver-specific expression of desired coding sequences beyond the current use with Factor IX.

国家/地区

美国