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Article

In vivo activation of the human δ-globin gene: the therapeutic potential in β-thalassemic mice.

Haematologica - 1 Jan 2014

Manchinu Maria F, Marongiu Maria F, Poddie Daniela, Casu Carla, Latini Veronica, Simbula Michela, Galanello Renzo, Moi Paolo, Cao Antonio, Porcu Susanna, Ristaldi Maria S

Abstract excerpt

β-thalassemia and sickle cell disease are widespread fatal genetic diseases. None of the existing clinical treatments provides a solution for all patients. Two main strategies for treatment are currently being investigated: (i) gene transfer of a normal β-globin gene; (ii) reactivation of the endogenous γ-globin gene. To date, neither approach has led to a satisfactory, commonly accepted standard of care. The...

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Identifiers

PMID: 23872310
DOI: 10.3324/haematol.2012.082768
PMCID: PMC4007936

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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