Decision-making about gene therapy in transfusion dependent thalassemia

Decision-making about gene therapy in transfusion dependent thalassemia

Abstract 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. Transplantat...

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Bibliographic Details
Main Authors: Maa-Ohui Quarmyne, Diana Ross, Cynthia Sinha, Nitya Bakshi, Jeanne Boudreaux, Lakshmanan Krishnamurti
Format: Article
Language:English
Published: BMC 2022-09-01
Series:BMC Pediatrics
Subjects:
Decision-making
Transfusion dependent thalassemia
Gene therapy
Qualitative
Online Access:https://doi.org/10.1186/s12887-022-03598-3

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