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.
gene therapy, hemophilia, factor IX
Granted Patents or Published Applications
US Patent No. 8,030,065; 8,168,425; Multiple foreign rights
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).
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.
Contact the Office of Technology Licensing (Phone: 901-595-2342, Fax: 901-595-3148) for more information.