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Preclinical evaluation for engraftment of CD34(+) cells gene-edited at the sickle cell disease locus in xenograft mouse and non-human primate models
Sickle cell disease (SCD) is caused by a 20A > T mutation in the β-globin gene. Genome-editing technologies have the potential to correct the SCD mutation in hematopoietic stem cells (HSCs), producing adult hemoglobin while simultaneously eliminating sickle hemoglobin. Here, we developed high-eff...
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| Publicado en: | Cell Rep Med |
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| Autores principales: | , , , , , , , , , , , , , , , , , |
| Formato: | Artigo |
| Lenguaje: | Inglês |
| Publicado: |
Elsevier
2021
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| Materias: | |
| Acceso en línea: | https://ncbi.nlm.nih.gov/pmc/articles/PMC8080237/ https://ncbi.nlm.nih.gov/pubmed/33948577 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.xcrm.2021.100247 |
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