An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype

An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype

Autologous transplantation of hematopoietic stem cells transduced with a lentiviral vector (LV) expressing an anti-sickling HBB variant is a potential treatment for sickle cell disease (SCD). With a clinical trial as our ultimate goal, we generated LV constructs containing an anti-sickling HBB trans...

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Bibliographic Details
Main Authors: Leslie Weber, Valentina Poletti, Elisa Magrin, Chiara Antoniani, Samia Martin, Charles Bayard, Hanem Sadek, Tristan Felix, Vasco Meneghini, Michael N. Antoniou, Wassim El-Nemer, Fulvio Mavilio, Marina Cavazzana, Isabelle Andre-Schmutz, Annarita Miccio
Format: Article
Language:English
Published: Elsevier 2018-09-01
Series:Molecular Therapy: Methods & Clinical Development
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050118300767

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