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Updated: Jun 14, 2026

In Vivo Osteo-organoid Approach for Harvesting Therapeutic Hematopoietic Stem/Progenitor Cells
Published on: February 16, 2024
Hematopoietic stem cell transplantation for hemoglobinopathies: current practice and emerging trends
Frans J Smiers1, Lakshmanan Krishnamurti, Guido Lucarelli
1Leiden University Medical Center, Department of Pediatrics, Hematology Oncology and BMT unit, Postbus 9600, 2300 RC, Leiden, The Netherlands. f.j.smiers@lumc.nl
Abstract:
Despite improvements in the management of thalassemia major and sickle cell disease, treatment complications are frequent and life expectancy remains diminished for these patients. Hematopoietic stem cell transplantation (HSCT) is the only curative option currently available. Existing results for HSCT in patients with hemoglobinopathy are excellent and still improving. New conditioning regimens are being used to reduce treatment-related toxicity and new donor pools accessed to increase the number of patients who can undergo HSCT.
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