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Limitations of mouse models for sickle cell disease conferred by their human globin transgene configurations

1 Jun 2022

Abstract excerpt

We characterized the human β-like globin transgenes in two mouse models of sickle cell disease (SCD) and tested a genome-editing strategy to induce red blood cell fetal hemoglobin (HbF; α2γ2). Berkeley SCD mice contain four to 22 randomly arranged, fragmented copies of three human transgenes (HBA1, HBG2-HBG1-HBD-HBBS and a mini-locus control region) integrated into a single site of mouse chromosome 1. Cas9...

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PMID: 35793591
DOI: 10.1242/dmm.049463

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