Decision-making about gene therapy in transfusion dependent thalassemia

• Main Authors: Maa-Ohui Quarmyne (Author), Diana Ross (Author), Cynthia Sinha (Author), Nitya Bakshi (Author), Jeanne Boudreaux (Author), Lakshmanan Krishnamurti (Author)
• Format: Book
• Published: BMC, 2022-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...