Ontology highlight
ABSTRACT:
SUBMITTER: Woodard KJ
PROVIDER: S-EPMC9277148 | biostudies-literature | 2022 Jun
REPOSITORIES: biostudies-literature
Woodard Kaitly J KJ Doerfler Phillip A PA Mayberry Kalin D KD Sharma Akshay A Levine Rachel R Yen Jonathan J Valentine Virginia V Palmer Lance E LE Valentine Marc M Weiss Mitchell J MJ
Disease models & mechanisms 20220601 6
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 disruption of the BCL11A repressor binding motif in the γ-globin gene (HBG1 and HBG2; HBG) ...[more]