Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease

Sickle cell disease (SCD) is caused by a mutation (E6V) in the hemoglobin (Hb) β-chain that induces polymerization of Hb tetramers, red blood cell deformation, ischemia, anemia, and multiple organ damage. Gene therapy is a potential alternative to human leukocyte antigen (HLA)-matched allogeneic hem...

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Detalhes bibliográficos
Publicado no:Mol Ther Methods Clin Dev
Main Authors: Poletti, Valentina, Urbinati, Fabrizia, Charrier, Sabine, Corre, Guillaume, Hollis, Roger P., Campo Fernandez, Beatriz, Martin, Samia, Rothe, Michael, Schambach, Axel, Kohn, Donald B., Mavilio, Fulvio
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2018
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6276308/
https://ncbi.nlm.nih.gov/pubmed/30533448
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2018.10.014
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