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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שמור ב:
מידע ביבליוגרפי
הוצא לאור ב:Cell Rep Med
Main Authors: 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.
פורמט: Artigo
שפה:Inglês
יצא לאור: Elsevier 2021
נושאים:
Article
גישה מקוונת: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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