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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...
Autores principales: | Sjeklocha, Lucas M., Park, Chang-Won, Wong, Phillip Y-P, Roney, Mark J., Belcher, John D., Kaufman, Dan S., Vercellotti, Gregory M., Hebbel, Robert P., Steer, Clifford J. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Public Library of Science
2011
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3247234/ https://www.ncbi.nlm.nih.gov/pubmed/22216176 http://dx.doi.org/10.1371/journal.pone.0029110 |
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