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Article

A preclinical approach for gene therapy of beta-thalassemia.

Annals of the New York Academy of Sciences - 1 Aug 2010

Breda Laura, Kleinert Dorothy A, Casu Carla, Casula Laura, Cartegni Luca, Fibach Eitan, Mancini Irene, Giardina Patricia J, Gambari Roberto, Rivella Stefano

Abstract excerpt

Lentiviral-mediated beta-globin gene transfer successfully treated beta-thalassemic mice. Based on this result, clinical trials were initiated. To date, however, no study has investigated the efficacy of gene therapy in relation to the nature of the different beta-globin mutations found in patien...

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Identifiers

PMID: 20712784
DOI: 10.1111/j.1749-6632.2010.05594.x
PMCID: PMC3068625

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 2 Sept 2026. Prepared record publication-20712784-2026-09-02.2. Manifest publication-graph-cohort-2026-09-02.2.

Source receipt: PubMed via Helena Literature Corpus, retrieved 2 Sept 2026.

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