Out-licensing

Robust Factor IX Minigene Expression Cassette: Gene Therapy for Inserting Genes that Require Liver-specific Expression

Researchers in Dr. Mark Kay's laboratory at Stanford University have designed a new liver-specific expression cassette for inserting genes into double-stranded AAV (adeno-associated virus) vectors for gene therapy. AAV vectors are useful for gene therapy because they can be used to transduce different types of dividing and non-dividing cells of different tissues. They can establish stable, long-term transgene expression without the toxicity or human diseases associated with other viral vectors. It is believed that double-stranded AAV vectors are 10 - 100 times more robust than single stranded AAV vectors and would therefore require much smaller doses than single-stranded AAV to produce a therapeutic effect. However, a limited amount of exogenous DNA can be inserted into the double-stranded vectors. Now, Stanford inventors have linked a synthetic liver-specific enhancer to a mini promoter to generate a Factor IX vector that can be packaged into double-stranded AAV vector. This cassette was used to express Factor IX in vivo.

Conclusion

The inventors have used the expression cassette with double stranded AAV vectors for Factor IX gene therapy in mice.

Relevance/Opportunity

We are currently seeking licensing or codevelopment partnerships. Please enquire quoting reference no. S06-099.

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