In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of β-thalassemia

Gene therapy for β-thalassemia requires stable transfer of a β-globin gene into hematopoietic stem cells (HSCs) and high and regulated hemoglobin expression in the erythroblastic progeny. We developed an erythroid-specific lentiviral vector driving the expression of the human β-globin gene from a mi...

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Detalhes bibliográficos
Main Authors: Miccio, Annarita, Cesari, Rossano, Lotti, Francesco, Rossi, Claudia, Sanvito, Francesca, Ponzoni, Maurilio, Routledge, Samantha J. E., Chow, Cheok-Man, Antoniou, Michael N., Ferrari, Giuliana
Formato: Artigo
Idioma:Inglês
Publicado em: National Academy of Sciences 2008
Assuntos:
Biological Sciences
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2492493/
https://ncbi.nlm.nih.gov/pubmed/18650378
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.0711666105
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