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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Udgivet i: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.
Format: Artigo
Sprog:Inglês
Udgivet: Elsevier 2021
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Article
Online adgang: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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