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Hematopoietic stem cell (HSC) gene therapy is potentially curative for sickle cell disease (SCD); 1 however, options for HSC collection are limited in this population, 2-4 and investigation of the collection, efficiency, and safety of peripheral blood (PB) mobilization with plerixafor from start to finish is needed. Here we describe consistent, safe, and sufficient PB HSC collection and processing after plerixafor mobilization from the greatest number of participants reported to date and the first twoinstitutional study. Our data suggest plerixafor mobilized HSC in SCD are enriched for long-term engrafting HSC, which is not true of HSC from SCD bone marrow (BM), 5 supporting a paradigm shift in the optimal HSC source for patients with SCD.
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Uchida et al. (2020) studied this question.
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