Preclinical evaluation of efficacy and safety of an improved lentiviral vector for the treatment of β-thalassemia and sickle cell disease

A previously published clinical trial demonstrated the benefit of autologous CD34[+] cells transduced with a self-inactivating lentiviral vector (HPV569) containing an engineered β-globin gene (β[A-T87Q] -globin) in a subject with β-thalassemia ...

Bibliographische Detailangaben

Hauptverfasser:	Negre, Olivier (VerfasserIn) , Bartholomä, Cynthia C. (VerfasserIn) , Deichmann, Annette (VerfasserIn) , Fronza, Raffaele (VerfasserIn) , Schmidt, Manfred (VerfasserIn) , Kalle, Christof von (VerfasserIn)
Dokumenttyp:	Article (Journal)
Sprache:	Englisch
Veröffentlicht:	2015 Feb.
In:	Current gene therapy Year: 2015, Jahrgang: 15, Heft: 1, Pages: 64-81
ISSN:	1875-5631
DOI:	10.2174/1566523214666141127095336
Online-Zugang:	Verlag, Volltext: https://doi.org/10.2174/1566523214666141127095336 Verlag, Volltext: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4440358/
Verfasserangaben:	Olivier Negre, Cynthia Bartholomae, Yves Beuzard, Marina Cavazzana, Lauryn Christiansen, Céline Courne, Annette Deichmann, Maria Denaro, Edouard de Dreuzy, Mitchell Finer, Raffaele Fronza, Béatrix Gillet-Legrand, Christophe Joubert, Robert Kutner, Philippe Leboulch, Leïla Maouche, Anaïs Paulard, Francis J. Pierciey, Jr, Michael Rothe, Byoung Ryu, Manfred Schmidt, Christof von Kalle, Emmanuel Payen, and Gabor Veres