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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| Hoofdauteurs: | , , , , , , , , |
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| Formaat: | Artigo |
| Taal: | Inglês |
| Gepubliceerd in: |
Public Library of Science (PLoS)
2011-01-01
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| Reeks: | PLoS ONE |
| Online toegang: | http://europepmc.org/articles/PMC3247234?pdf=render |
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