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At least 20% donor myeloid chimerism is necessary to reverse the sickle phenotype after allogeneic HSCT

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 al...

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Publicat a:Blood
Autors principals: Fitzhugh, Courtney D., Cordes, Stefan, Taylor, Tiffani, Coles, Wynona, Roskom, Katherine, Link, Mary, Hsieh, Matthew M., Tisdale, John F.
Format: Artigo
Idioma:Inglês
Publicat: American Society of Hematology 2017
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC5659067/
https://ncbi.nlm.nih.gov/pubmed/28887325
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2017-03-772392
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