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Using gene transfer to circumvent off-target effects

Gene Therapy volume 15, pages 759–764 (2008)Cite this article

Abstract

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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Authors and Affiliations

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

    C P Miller & C A Blau

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  1. C P Miller
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  2. C A Blau
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Correspondence to C A Blau.

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Miller, C., Blau, C. Using gene transfer to circumvent off-target effects. Gene Ther 15, 759–764 (2008). https://doi.org/10.1038/gt.2008.43

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