Using gene transfer to circumvent off-target effects
CP Miller, CA Blau - Gene therapy, 2008 - nature.com
CP Miller, CA Blau
Gene therapy, 2008•nature.comMany 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.
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.
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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