Hemophilia

In Vitro Success Does Not Directly Correspond With In Vivo Success

Published in Gene Therapy Weekly, March 25th, 1996

Cells genetically engineered to produce the therapeutic protein factor IX using the Baxter TheraCyte^TM have reverse efficiencies in vivo as compared to in vitro expression studies.

Gloria H. Frost et al. presented this surprising data at the Keystone Symposia on Tissue Engineering and Wound Repair in Context, held January 23-29, 1996 in Taos, New Mexico.

The authors noted that for successful ex vivo gene therapy, the optimal genetically engineered cell is one that is stable and produces high amounts of a therapeutic protein. In vitro expression data is used to determine which gene therapy protocol should be most effective in vivo.

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Source: Gene Therapy Weekly (1996-03-25)

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