Integration-deficient Lentiviral Vectors Expressing Codon-optimized R338L Human FIX Restore Normal Hemostasis in Hemophilia B Mice

Integration-deficient lentiviral vectors (IDLVs) have been shown to transduce a wide spectrum of target cells and organs in vitro and in vivo and to maintain long-term transgene expression in nondividing cells. However, epigenetic silencing of episomal vector genomes reduces IDLV transgene expressio...

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Bibliographische Detailangaben
Hauptverfasser: Suwanmanee, Thipparat, Hu, Genlin, Gui, Tong, Bartholomae, Cynthia C, Kutschera, Ina, von Kalle, Christof, Schmidt, Manfred, Monahan, Paul E, Kafri, Tal
Format: Artigo
Sprache:Inglês
Veröffentlicht: Nature Publishing Group 2014
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Original Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3944325/
https://ncbi.nlm.nih.gov/pubmed/23941813
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2013.188
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