Comparative targeting analysis of KLF1, BCL11A, and HBG1/2 in CD34⁺ HSPCs by CRISPR/Cas9 for the induction of fetal hemoglobin.

ABSTRACT: β-hemoglobinopathies are caused by abnormal or absent production of hemoglobin in the blood due to mutations in the β-globin gene (HBB). Imbalanced expression of adult hemoglobin (HbA) induces strong anemia in patients suffering from the disease. However, individuals with natural-occurring mutations in the HBB cluster or related genes, compensate this disparity through γ-globin expression and subsequent fetal hemoglobin (HbF) production. Several preclinical and clinical studies have been performed in order to induce HbF by knocking-down genes involved in HbF repression (KLF1 and BCL11A) or disrupting the binding sites of several transcription factors in the γ-globin gene (HBG1/2). In this study, we thoroughly compared the different CRISPR/Cas9 gene-disruption strategies by gene editing ana

SUBMITTER: Lamsfus-Calle A 

PROVIDER: S-EPMC7311455 | biostudies-literature | 2020 Jun

REPOSITORIES: biostudies-literature