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Permanent and panerythroid correction of murine β thalassemia by multiple lentiviral integration in hematopoietic stem cells

Achieving long-term pancellular expression of a transferred gene at therapeutic level in a given hematopoietic lineage remains an important goal of gene therapy. Advances have recently been made in the genetic correction of the hemoglobinopathies by means of lentiviral vectors and large locus contro...

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Main Authors: Imren, Suzan, Payen, Emmanuel, Westerman, Karen A., Pawliuk, Robert, Fabry, Mary E., Eaves, Connie J., Cavilla, Benjamin, Wadsworth, Louis D., Beuzard, Yves, Bouhassira, Eric E., Russell, Robert, London, Irving M., Nagel, Ronald L., Leboulch, Philippe, Humphries, R. Keith
Formato: Artigo
Idioma:Inglês
Publicado: National Academy of Sciences 2002
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Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC137892/
https://ncbi.nlm.nih.gov/pubmed/12391330
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.212507099
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