Therapeutic hemoglobin levels after gene transfer in β-thalassemia mice and in hematopoietic cells of β-thalassemia and sickle cells disease patients.

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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Bibliographic Details
Main Authors: Laura Breda, Carla Casu, Sara Gardenghi, Nicoletta Bianchi, Luca Cartegni, Mohandas Narla, Karina Yazdanbakhsh, Marco Musso, Deepa Manwani, Jane Little, Lawrence B Gardner, Dorothy A Kleinert, Eugenia Prus, Eitan Fibach, Robert W Grady, Patricia J Giardina, Roberto Gambari, Stefano Rivella
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2012-01-01
Series:PLoS ONE
Online Access:http://europepmc.org/articles/PMC3314006?pdf=render

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