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Knowledge to date on secondary malignancy following hematopoietic cell transplantation for sickle cell disease

9 Dec 2022

Abstract excerpt

Allogeneic hematopoietic cell transplantation, gene therapy, and gene editing offer a potential cure for sickle cell disease (SCD). Unfortunately, myelodysplastic syndrome and acute myeloid leukemia development have been higher than expected after graft rejection following nonmyeloablative conditioning and lentivirus-based gene therapy employing myeloablative busulfan for SCD. Somatic mutations discovered in 2 of...

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PMID: 36485129
DOI: 10.1182/hematology.2022000371

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