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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izdano v:Mol Ther Methods Clin Dev
Main Authors: Weber, Leslie, Poletti, Valentina, Magrin, Elisa, Antoniani, Chiara, Martin, Samia, Bayard, Charles, Sadek, Hanem, Felix, Tristan, Meneghini, Vasco, Antoniou, Michael N., El-Nemer, Wassim, Mavilio, Fulvio, Cavazzana, Marina, Andre-Schmutz, Isabelle, Miccio, Annarita
Format: Artigo
Jezik:Inglês
Izdano: American Society of Gene & Cell Therapy 2018
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Article
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC6105766/
https://ncbi.nlm.nih.gov/pubmed/30140714
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2018.07.012
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