Safety and efficacy of plerixafor dose escalation for the mobilization of CD34(+) hematopoietic progenitor cells in patients with sickle cell disease: interim results

Gene therapy for sickle cell disease is limited by the yield of hematopoietic progenitor cells that can be harvested for transduction or gene editing. We therefore performed a phase I dose-escalation study of the hematopoietic progenitor cell mobilizing agent plerixafor to evaluate the efficacy and...

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Veröffentlicht in:Haematologica
Hauptverfasser: Boulad, Farid, Shore, Tsiporah, van Besien, Koen, Minniti, Caterina, Barbu-Stevanovic, Mihaela, Fedus, Sylvie Wiener, Perna, Fabiana, Greenberg, June, Guarneri, Danielle, Nandi, Vijay, Mauguen, Audrey, Yazdanbakhsh, Karina, Sadelain, Michel, Shi, Patricia A.
Format: Artigo
Sprache:Inglês
Veröffentlicht: Ferrata Storti Foundation 2018
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Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5927989/
https://ncbi.nlm.nih.gov/pubmed/29419425
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3324/haematol.2017.187047
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