Review Article
Gene Therapy (2008) 15, 759–764; doi:10.1038/gt.2008.43; published online 20 March 2008
Using gene transfer to circumvent off-target effects
1Department of Medicine/Hematology, Institute for Stem Cell and Regenerative Medicine, University of Washington, Seattle, WA, USA
Correspondence: Dr CA Blau, Department of Medicine/Hematology, University of Washington, K260, Health Science Building, Mailstop 357710 Seattle, Washington, WA 98195, USA. E-mail: tblau@u.washington.edu
Received 18 February 2008; Accepted 18 February 2008; Published online 20 March 2008.
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.
Keywords:
erythropoietin, cancer, regulation, dimerizer
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