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Regioselective Biolistic Targeting in Organotypic Brain Slices Using a Modified Gene Gun
Published on: October 24, 2014
Using gene transfer to circumvent off-target effects
1Department of Medicine/Hematology, Institute for Stem Cell and Regenerative Medicine, University of Washington, Seattle, WA 98195, USA.
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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