Erythroid-specific Human Factor IX Delivery From In Vivo Selected Hematopoietic Stem Cells Following Nonmyeloablative Conditioning in Hemophilia B Mice

We have developed a lentiviral vector system for human factor IX (hFIX) gene transfer in hematopoietic stem cells (HSCs) that provides erythroid cell-derived systemic protein delivery following nonmyeloablative conditioning and in vivo methylguanine methyltransferase (MGMT) drug selection. After bon...

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Main Authors: Chang, Alex H, Stephan, Matthias T, Lisowski, Leszek, Sadelain, Michel
Formato: Artigo
Idioma:Inglês
Publicado: 2008
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Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC2658893/
https://ncbi.nlm.nih.gov/pubmed/18682698
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2008.161
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