A combined approach for β-thalassemia based on gene therapy-mediated adult hemoglobin (HbA) production and fetal hemoglobin (HbF) induction

Gene therapy might fall short in achieving a complete reversion of the β-thalassemic phenotype due to current limitations in vector design and myeloablative regimen. Following gene transfer, all or a large proportion of erythroid cells might express suboptimal levels of β-globin, impairing the thera...

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Hauptverfasser: Zuccato, Cristina, Breda, Laura, Salvatori, Francesca, Breveglieri, Giulia, Gardenghi, Sara, Bianchi, Nicoletta, Brognara, Eleonora, Lampronti, Ilaria, Borgatti, Monica, Rivella, Stefano, Gambari, Roberto
Format: Artigo
Sprache:Inglês
Veröffentlicht: Springer-Verlag 2012
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Original Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3389244/
https://ncbi.nlm.nih.gov/pubmed/22460946
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s00277-012-1430-5
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