Therapeutic Hemoglobin Levels after Gene Transfer in β-Thalassemia Mice and in Hematopoietic Cells of β-Thalassemia and Sickle Cells Disease Patients

Preclinical and clinical studies demonstrate the feasibility of treating β-thalassemia and Sickle Cell Disease (SCD) by lentiviral-mediated transfer of the human β-globin gene. However, previous studies have not addressed whether the ability of lentiviral vectors to increase hemoglobin synthesis mig...

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Detalles Bibliográficos
Autores principales: Breda, Laura, Casu, Carla, Gardenghi, Sara, Bianchi, Nicoletta, Cartegni, Luca, Narla, Mohandas, Yazdanbakhsh, Karina, Musso, Marco, Manwani, Deepa, Little, Jane, Gardner, Lawrence B., Kleinert, Dorothy A., Prus, Eugenia, Fibach, Eitan, Grady, Robert W., Giardina, Patricia J., Gambari, Roberto, Rivella, Stefano
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2012
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3314006/
https://www.ncbi.nlm.nih.gov/pubmed/22479321
http://dx.doi.org/10.1371/journal.pone.0032345

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