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 ...
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| Hauptverfasser: | , , , , , |
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| Dokumenttyp: | Article (Journal) |
| Sprache: | Englisch |
| Veröffentlicht: |
2015 Feb.
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| 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/ 
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| 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 |
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