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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.


C P Miller, C A Blau. Using gene transfer to circumvent off-target effects. Gene therapy. 2008 May;15(10):759-64

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PMID: 18356813

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