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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...
Autores principales: | 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. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Ferrata Storti Foundation
2018
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5927989/ https://www.ncbi.nlm.nih.gov/pubmed/29419425 http://dx.doi.org/10.3324/haematol.2017.187047 |
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