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Hematopoietic stem cell collection for sickle cell disease gene therapy

9 Feb 2024

Abstract excerpt

PURPOSE OF REVIEW: Gene therapy for sickle cell disease (SCD) is advancing rapidly, with two transformative products recently approved by the US Food and Drug Administration and numerous others under study. All current gene therapy protocols require ex vivo modification of autologous hematopoietic stem cells (HSCs). However, several SCD-related problems impair HSC collection, including a stressed and damaged bone...

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PMID: 38359264
DOI: 10.1097/moh.0000000000000807

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