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Article

Correction of phenotype in a thalassemia mouse model using a nonmyeloablative marrow transplantation regimen.

Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation - 1 Jan 2002

Bradley M Brigid, Sattler Rose M, Raftopoulos Harry, Ward Maureen, Grossman I Robert, Townes Tim M, Ryan Thomas A, Bank Arthur

Abstract excerpt

Gene therapy, the replacement of normal human beta- or gamma-globin genes into the hematopoietic stem cells of patients with homozygous beta-thalassemia, is a promising therapy for the future. High-level lineage-specific stable globin expression in transduced cells reinfused into patients in an autologous transplantation setting could be curative, if successful. Previous studies have shown high-level donor...

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Identifiers

PMID: 12234171
DOI: 10.1053/bbmt.2002.v8.pm12234171

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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