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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...
Autores principales: | , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group
2015
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4667717/ https://www.ncbi.nlm.nih.gov/pubmed/26665131 http://dx.doi.org/10.1038/mtm.2015.45 |