Minimized Liver Specific Promoter for Improved Gene Expression in Gene Therapy Vectors (SJ-04-0024)

St. Jude Reference #SJ-04-0024

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.


 

Keywords

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).


 

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.

Related Links

Contact the Office of Technology Licensing (Phone: 901-595-2342, Fax: 901-595-3148) for more information.