title: Using gene transfer to circumvent off-target effects.
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.
authors: Department of Medicine/Hematology, Institute for Stem Cell and Regenerative Medicine, University of Washington, Seattle, WA 98195, USA.
authors: C P
authors: CP
authors: Miller
authors: C A
authors: CA
authors: Blau
mesh_terms: D000740:Anemia / Q000097:blood / Q000628:therapy; D000818:Animals; D049109:Cell Proliferation; D017690:Cell Transplantation / Q000379:methods*; D005786:Gene Expression Regulation; D005818:Genetic Engineering; D015316:Genetic Therapy / Q000379:methods*; D006410:Hematopoiesis; D006801:Humans; D009369:Neoplasms / Q000097:blood / Q000628:therapy*; D009930:Organic Chemicals / Q000494:pharmacology; D017467:Receptors, Erythropoietin / Q000235:genetics / Q000378:metabolism; D016559:Tacrolimus / Q000031:analogs & derivatives / Q000494:pharmacology / Q000627:therapeutic use
chemical_list: C084260:FK 1012; D009930:Organic Chemicals; D017467:Receptors, Erythropoietin; C423866:AP 1903 reagent; D016559:Tacrolimus