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Article

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

PloS one - 1 Jan 2012

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

Abstract excerpt

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...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 22479321
DOI: 10.1371/journal.pone.0032345
PMCID: PMC3314006

Genes

SCD

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 4 Sept 2026. Prepared record publication-22479321-2026-09-04.2. Manifest publication-graph-cohort-2026-09-04.2.

Source receipt: PubMed via Helena Literature Corpus, retrieved 4 Sept 2026.

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