Bone Marrow as a Hematopoietic Stem Cell Source for Gene Therapy in Sickle Cell Disease: Evidence from Rhesus and SCD Patients

Steady state bone marrow (BM) is the preferred hematopoietic stem cell (HSC) source for gene therapy in sickle cell disease (SCD) due to the recognized risk of vaso-occlusive crisis during granulocyte colony–stimulating factor mobilization. We previously established clinically relevant HSC gene tran...

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Udgivet i:Hum Gene Ther Clin Dev
Main Authors: Uchida, Naoya, Fujita, Atsushi, Hsieh, Matthew M., Bonifacino, Aylin C., Krouse, Allen E., Metzger, Mark E., Donahue, Robert E., Tisdale, John F.
Format: Artigo
Sprog:Inglês
Udgivet: Mary Ann Liebert, Inc. 2017
Fag:
Research Articles
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5695729/
https://ncbi.nlm.nih.gov/pubmed/28447889
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/humc.2017.029
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