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
Autores principales: Uchida, Naoya, Li, Linhong, Nassehi, Tina, Drysdale, Claire M., Yapundich, Morgan, Gamer, Jackson, Haro-Mora, Juan J., Demirci, Selami, Leonard, Alexis, Bonifacino, Aylin C., Krouse, Allen E., Linde, N. Seth, Allen, Cornell, Peshwa, Madhusudan V., De Ravin, Suk See, Donahue, Robert E., Malech, Harry L., Tisdale, John F.
Formato: Artigo
Lenguaje:Inglês
Publicado: Elsevier 2021
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Article
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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