Erythroid-specific Expression of β-globin by Sleeping Beauty Transposon for Sickle Cell Disease

Sickle cell disease (SCD) results predominately from a single monogenic mutation that affects thousands of individuals worldwide. Gene therapy approaches have focused on using viral vectors to transfer wild type β- or γ-globin transgenes into hematopoietic stem cells for long-term expression of the...

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Bibliographic Details
Main Authors: Zhu, Jianhui, Kren, Betsy T., Park, Chang Won, Bilgim, Rasim, Wong, Phillip Y-P., Steer, Clifford J.
Format: Artigo
Language:Inglês
Published: 2007
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Article
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC3893920/
https://ncbi.nlm.nih.gov/pubmed/17508724
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1021/bi6024484
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