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 major. This vector has been modified to increase transdu...

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Publicat a:Curr Gene Ther
Autors principals: Negre, Olivier, Bartholomae, Cynthia, Beuzard, Yves, Cavazzana, Marina, Christiansen, Lauryn, Courne, Céline, Deichmann, Annette, Denaro, Maria, de Dreuzy, Edouard, Finer, Mitchell, Fronza, Raffaele, Gillet-Legrand, Béatrix, Joubert, Christophe, Kutner, Robert, Leboulch, Philippe, Maouche, Leïla, Paulard, Anaïs, Pierciey, Francis J., Rothe, Michael, Ryu, Byoung, Schmidt, Manfred, von Kalle, Christof, Payen, Emmanuel, Veres, Gabor
Format: Artigo
Idioma:Inglês
Publicat: Bentham Science Publishers 2015
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Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC4440358/
https://ncbi.nlm.nih.gov/pubmed/25429463
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.2174/1566523214666141127095336
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