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Erythroid-specific expression of β-globin from Sleeping Beauty-transduced human hematopoietic progenitor cells.

Gene therapy for sickle cell disease will require efficient delivery of a tightly regulated and stably expressed gene product to provide an effective therapy. In this study we utilized the non-viral Sleeping Beauty (SB) transposon system using the SB100X hyperactive transposase to transduce human co...

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Main Authors: Lucas M Sjeklocha, Chang-Won Park, Phillip Y-P Wong, Mark J Roney, John D Belcher, Dan S Kaufman, Gregory M Vercellotti, Robert P Hebbel, Clifford J Steer
Format: Artigo
Language:Inglês
Published: Public Library of Science (PLoS) 2011-01-01
Series:PLoS ONE
Online Access:http://europepmc.org/articles/PMC3247234?pdf=render
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