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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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Detalles Bibliográficos
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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2011
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