Article
At least 20% donor myeloid chimerism is necessary to reverse the sickle phenotype after allogeneic HSCT.
Blood - 26 Oct 2017
Fitzhugh Courtney D, Cordes Stefan, Taylor Tiffani, Coles Wynona, Roskom Katherine, Link Mary, Hsieh Matthew M, Tisdale John F
Abstract excerpt
Novel curative therapies using genetic transfer of normal globin-producing genes into autologous hematopoietic stem cells (HSCs) are in clinical trials for patients with sickle cell disease (SCD). The percentage of transferred globin necessary to cure SCD is currently not known. In the setting of allogeneic nonmyeloablative HSC transplants (HSCTs), stable mixed chimerism is sufficient to reverse the disease. We...
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