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Implications of hematopoietic stem cells heterogeneity for gene therapies

15 Feb 2021

Abstract excerpt

Hematopoietic stem cell transplantation (HSCT) is the therapeutic concept to cure the blood/immune system of patients suffering from malignancies, immunodeficiencies, red blood cell disorders, and inherited bone marrow failure syndromes. Yet, allogeneic HSCT bear considerable risks for the patient such as non-engraftment, or graft-versus host disease. Transplanting gene modified autologous HSCs is a promising...

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PMID: 33589780
DOI: 10.1038/s41434-021-00229-x

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