Minimized Liver Specific Promoter for Improved Gene Expression in Gene Therapy Vectors (SJ-04-0024 & SJ-06-0008)





Technology Name
(St. Jude Reference #)

Minimized Liver Specific Promoter forImproved Gene Expression in Gene Therapy Vectors
(SJ-04-0024 & SJ-06-0008)

Description Improving expression levels is a key for making gene therapy work in humans.  A liver specific promoter with regulatory regions minimized in size is provided.  This promoter can be 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
Granted Patents or Published Applications U.S. Appln. No. 2008/0153156; pending European application
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. 

 

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Last update: September 2008