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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Vydáno v:Cell Rep Med
Hlavní autoři: 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.
Médium: Artigo
Jazyk:Inglês
Vydáno: Elsevier 2021
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Article
On-line přístup: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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