Article

Advances in stem cell transplantation and gene therapy in the β-hemoglobinopathies

2012-12-08

Abstract excerpt

Abstract High-level production of β-globin, γ-globin, or therapeutic mutant globins in the RBC lineage by hematopoietic stem cell gene therapy ameliorates or cures the hemoglobinopathies sickle cell disease and beta thalassemia, which are major causes of morbidity and mortality worldwide. Considerable efforts have been made in the last 2 decades in devising suitable gene-transfer vectors and protocols to achieve t...

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Literature Corpus work: c726cb12-3158-53fd-aadb-3b361597b9fd
DOI: 10.1182/asheducation.v2012.1.276.3807841

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