Safe mobilization of CD34(+) cells in adults with β-thalassemia and validation of effective globin gene transfer for clinical investigation

We conducted a pilot trial to investigate the safety and effectiveness of mobilizing CD34(+) hematopoietic progenitor cells (HPCs) in adults with β-thalassemia major. We further assessed whether thalassemia patient CD34(+) HPCs could be transduced with a globin lentiviral vector under clinical condi...

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Autores principales: Boulad, Farid, Wang, Xiuyan, Qu, Jinrong, Taylor, Clare, Ferro, Leda, Karponi, Garyfalia, Bartido, Shirley, Giardina, Patricia, Heller, Glenn, Prockop, Susan E., Maggio, Aurelio, Sadelain, Michel, Rivière, Isabelle
Formato: Artigo
Lenguaje:Inglês
Publicado: American Society of Hematology 2014
Materias:
Gene Therapy
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC3945860/
https://ncbi.nlm.nih.gov/pubmed/24429337
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2013-06-507178
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