Ontology highlight
ABSTRACT:
SUBMITTER: Negre O
PROVIDER: S-EPMC4440358 | biostudies-literature | 2015
REPOSITORIES: biostudies-literature
Negre Olivier O Bartholomae Cynthia C Beuzard Yves Y Cavazzana Marina M Christiansen Lauryn L Courne Céline C Deichmann Annette A Denaro Maria M de Dreuzy Edouard E Finer Mitchell M Fronza Raffaele R Gillet-Legrand Béatrix B Joubert Christophe C Kutner Robert R Leboulch Philippe P Maouche Leïla L Paulard Anaïs A Pierciey Francis J FJ Rothe Michael M Ryu Byoung B Schmidt Manfred M von Kalle Christof C Payen Emmanuel E Veres Gabor G
Current gene therapy 20150101 1
A previously published clinical trial demonstrated the benefit of autologous CD34(+) cells transduced with a selfinactivating 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 transduction efficacy without compromising safety. In vitro analyses indicated that the changes resulted in both increased vector titers (3 to 4 fold) and increased transduction efficacy (2 to 3 fold). An in ...[more]