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Decision-making about gene therapy in transfusion dependent thalassemia

9 Sept 2022

Abstract excerpt

BACKGROUND: Hematopoietic stem cell transplantation (HSCT) is a treatment option with curative intent for patients with transfusion dependent thalassemia (TDT) but its application is limited by the lack of suitable donors and acceptability due to the related morbidity/mortality. Transplantation of autologous genetically modified hematopoietic cells, gene therapy (GT) is emerging as a promising treatment option...

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PMID: 36085025
DOI: 10.1186/s12887-022-03598-3

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