Amelioration of murine sickle cell disease by nonablative conditioning and γ-globin gene-corrected bone marrow cells

Amelioration of murine sickle cell disease by nonablative conditioning and γ-globin gene-corrected bone marrow cells

Patients with severe sickle cell disease (SCD) are candidates for gene therapy using autologous hematopoietic stem cells (HSCs), but concomitant multi-organ disease may contraindicate pretransplant conditioning with full myeloablation. We tested whether nonmyeloablative conditioning, a regimen used...

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Bibliographic Details
Main Authors: Tamara I Pestina, Phillip W Hargrove, Huifen Zhao, Paul E Mead, Matthew P Smeltzer, Mitchell J Weiss, Andrew Wilber, Derek A Persons
Format: Article
Language:English
Published: Elsevier 2015-01-01
Series:Molecular Therapy: Methods & Clinical Development
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050116300572

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