NDLI: Permanent and panerythroid correction of murine beta thalassemia by multiple lentiviral integration in hematopoietic stem cells.

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

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

Content Provider	World Health Organization (WHO)-Global Index Medicus
Author	Westerman, Karen A. Wadsworth, Louis D. Beuzard, Yves Bouhassira, Eric E. Imren, Suzan Cavilla, Benjamin Fabry, Mary E. London, Irving M. Eaves, Connie J. Russell, Robert Pawliuk, Robert Nagel, Ronald L. Payen, Emmanuel Humphries, R. Keith Leboulch, Philippe
Description	Author Affiliation: Imren S ( Terry Fox Laboratory, British Columbia Cancer Agency, Vancouver, BC, Canada.);
Abstract	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 control region (LCR) derivatives. However, panerythroid beta globin gene expression has not yet been achieved in beta thalassemic mice because of incomplete transduction of the hematopoietic stem cell compartment and position effect variegation of proviruses integrated at a single copy per genome. Here, we report the permanent, panerythroid correction of severe beta thalassemia in mice, resulting from a homozygous deletion of the beta major globin gene, by transplantation of syngeneic bone marrow transduced with an HIV-1-derived [beta globin gene/LCR] lentiviral vector also containing the Rev responsive element and the central polypurine tract/DNA flap. The viral titers produced were high enough to achieve transduction of virtually all of the hematopoietic stem cells in the graft with an average of three integrated proviral copies per genome in all transplanted mice; the transduction was sustained for >7 months in both primary and secondary transplants, at which time approximately 95% of the red blood cells in all mice contained human beta globin contributing to 32 +/- 4% of all beta-like globin chains. Hematological parameters approached complete phenotypic correction, as assessed by hemoglobin levels and reticulocyte and red blood cell counts. All circulating red blood cells became and remained normocytic and normochromic, and their density was normalized. Free alpha globin chains were completely cleared from red blood cell membranes, splenomegaly abated, and iron deposit was almost eliminated in liver sections. These findings indicate that virtually complete transduction of the hematopoietic stem cell compartment can be achieved by high-titer lentiviral vectors and that position effect variegation can be mitigated by multiple events of proviral integration to yield balanced, panerythroid expression. These results provide a solid foundation for the initiation of human clinical trials in beta thalassemia patients.
ISSN	00278424
e-ISSN	10916490
Journal	Proceedings of the National Academy of Sciences of the United States of America
Issue Number	22
Volume Number	99
Language	English
Publisher	National Academy of Sciences
Publisher Date	2002-10-01
Publisher Place	United States
Access Restriction	Open
Subject Keyword	Erythroid Precursor Cells Metabolism Genetic Vectors Globins Genetics Lentivirus Virus Integration Beta-Thalassemia Therapy 3T3 Cells Animals COS Cells Cercopithecus Aethiops Disease Models, Animal Erythrocytes Cytology Gene Expression Liver Pathology Mice Mice, Inbred C57BL Spleen Research Support, Non-U.S. Gov't Research Support, U.S. Gov't, P.H.S. Multidisciplinary
Content Type	Text
Resource Type	Article
Subject	Multidisciplinary

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