Potentially Therapeutic Levels of Anti-Sickling Globin Gene Expression Following Lentivirus-mediated Gene Transfer in Sickle Cell Disease Bone Marrow CD34(+) Cells

Sickle cell disease (SCD) can be cured by allogeneic hematopoietic stem cell (HSC) transplant. However, this is only possible when a matched donor is available making the development of gene therapy using autologous HSCs a highly desired alternative. We used a culture model of human erythropoiesis t...

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Publicado no:Exp Hematol
Main Authors: Urbinati, Fabrizia, Hargrove, Philip W., Geiger, Sabine, Romero, Zulema, Wherley, Jennifer, Kaufman, Michael L., Hollis, Roger P., Chambers, Christopher B., Persons, Derek A., Kohn, Donald B., Wilber, Andrew
Formato: Artigo
Idioma:Inglês
Publicado em: 2015
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4428920/
https://ncbi.nlm.nih.gov/pubmed/25681747
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.exphem.2015.01.009
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