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

doi:10.1073/pnas.0711666105

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Article

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

Proceedings of the National Academy of Sciences of the United States of America - 29 Jul 2008

Miccio Annarita, Cesari Rossano, Lotti Francesco, Rossi Claudia, Sanvito Francesca, Ponzoni Maurilio, Routledge Samantha J E, Chow Cheok-Man, Antoniou Michael N, Ferrari Giuliana

Abstract excerpt

Gene therapy for beta-thalassemia requires stable transfer of a beta-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 beta-globin gene from a minimal promoter/enhancer element containing two hypersensitive sites from the beta-globin locus control...

Topics

Animals
Cell Transplantation
Cloning, Molecular
Erythroblasts
Gene Expression Regulation
Genetic Therapy
Genetic Vectors
Globins
Hematopoietic Stem Cells

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Humans

Mice

Mice, Inbred C57BL

Phenotype

Stem Cells

beta-Thalassemia

Source

Open in PubMed Open full text

Identifiers

PMID: 18650378
DOI: 10.1073/pnas.0711666105
PMCID: PMC2492493

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