Preclinical Evaluation of Efficacy and Safety of an Improved Lentiviral Vector for the Treatment of &#946;-Thalassemia and Sickle Cell Disease

Negre, Olivier; Bartholomae, Cynthia; Beuzard, Yves; Cavazzana, Marina; Christiansen, Lauryn; Courne, Celine; Deichmann, Annette; Denaro, Maria; Dreuzy, Edouard; Finer, Mitchell; Fronza, Raffaele; Gillet-Legrand, Beatrix; Joubert, Christophe; Kutner, Robert; Leboulch, Philippe; Maouche, Leila; Paulard, Anais; Pierciey, Francis; Rothe, Michael; Ryu, Byoung; Schmidt, Manfred; Kalle, Christof; Payen, Emmanuel; Veres, Gabor

doi:10.2174/1566523214666141127095336

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 (βA-T87Q-globin) in a subject with β-thalassemia major. This vector has been modified to increase transduc…

Preclinical Evaluation of Efficacy and Safety of an Improved Lentiviral Vector for the Treatment of &#946;-Thalassemia and Sickle Cell Disease

Preclinical Evaluation of Efficacy and Safety of an Improved Lentiviral Vector for the Treatment of β-Thalassemia and Sickle Cell Disease