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Gene Ther. 2008 May;15(10):759-64. doi: 10.1038/gt.2008.43. Epub 2008 Mar 20.

Using gene transfer to circumvent off-target effects.

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  • 1Department of Medicine/Hematology, Institute for Stem Cell and Regenerative Medicine, University of Washington, Seattle, WA 98195, USA.


Many recombinant growth factors have failed in clinical trials due to off-target effects. We describe a method for circumventing off-target effects that involves equipping cells with a conditionally active signaling protein that can be specifically activated by an exogenously administered synthetic ligand. We believe that this approach will have many applications in gene and cell therapy.

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