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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Publicado en:Mol Ther Methods Clin Dev
Autores principales: Pestina, Tamara I, Hargrove, Phillip W, Zhao, Huifen, Mead, Paul E, Smeltzer, Matthew P, Weiss, Mitchell J, Wilber, Andrew, Persons, Derek A
Formato: Artigo
Lenguaje:Inglês
Publicado: Nature Publishing Group 2015
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Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC4667717/
https://ncbi.nlm.nih.gov/pubmed/26665131
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtm.2015.45
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