ArticlePloS one2019
CRISPR-Cas9 interrogation of a putative fetal globin repressor in human erythroid cells.
Article in PloS one, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.
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Who cites it
17 citing papers in PubMed, 26 citations in OpenAlex.
- Investigating the Efficacy of the CRISPR/Cas9 Gene-Editing System for Targeting the HBB FSC 36-37 (-T) Mutation Locus in Hematopoietic Stem Cells.Molecular biotechnology · 2026Article
- Complex HBB gene editing outcomes revealed by a fluorescent reporter cell model.Molecular therapy. Nucleic acids · 2026Article
- CRISPR/Cas-based gene editing in therapeutic strategies for beta-thalassemia.Human genetics · 2023Review
- Editing the core region in HPFH deletions alters fetal and adult globin expression for treatment of β-hemoglobinopathies.Molecular therapy. Nucleic acids · 2023Article
- Erythroid lineage-specific lentiviral RNAi vectors suitable for molecular functional studies and therapeutic applications.Scientific reports · 2022Article
- Article
- Heterozygosity of the Complex Corfu δ0β+ Thalassemic Allele (HBD Deletion and HBB:c.92+5G>A) RevisitedBiology · 2022Article
- Article
- Editing outside the body: Ex vivo gene-modification for β-hemoglobinopathy cellular therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2021Review
- Efficient CRISPR-Cas9-based genome editing of β-globin gene on erythroid cells from homozygous βMolecular therapy. Methods & clinical development · 2021Article
- Direct Generation of Immortalized Erythroid Progenitor Cell Lines from Peripheral Blood Mononuclear Cells.Cells · 2021Article
- Manipulation of Developmental Gamma-Globin Gene Expression: an Approach for Healing Hemoglobinopathies.Molecular and cellular biology · 2020Review
- Comparative targeting analysis of KLF1, BCL11A, and HBG1/2 in CD34Scientific reports · 2020Article
- Review
- β-Hemoglobinopathies: The Test Bench for Genome Editing-Based Therapeutic Strategies.Frontiers in genome editing · 2020Review
- Rosetting revisited: a critical look at the evidence for host erythrocyte receptors inParasitology · 2020Review
- Genome editing for blood disorders: state of the art and recent advances.Emerging topics in life sciences · 2019Article
Corrections and comments
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Authors and funding
12 authors at 5 institutions in 3 countries.
Funding
Abstract
Sickle Cell Disease and ß-thalassemia, which are caused by defective or deficient adult ß-globin (HBB) respectively, are the most common serious genetic blood diseases in the world. Persistent expression of the fetal ß-like globin, also known as 𝛾-globin, can ameliorate both disorders by serving in place of the adult ß-globin as a part of the fetal hemoglobin tetramer (HbF). Here we use CRISPR-Cas9 gene editing to explore a potential 𝛾-globin silencer region upstream of the δ-globin gene identified by comparison of naturally-occurring deletion mutations associated with up-regulated 𝛾-globin. We find that deletion of a 1.7 kb consensus element or select 350 bp sub-regions from bulk populations of cells increases levels of HbF. Screening of individual sgRNAs in one sub-region revealed three single guides that caused increases in 𝛾-globin expression. Deletion of the 1.7 kb region in HUDEP-2 clonal sublines, and in colonies derived from CD34+ hematopoietic stem/progenitor cells (HSPCs), does not cause significant up-regulation of 𝛾-globin. These data suggest that the 1.7 kb region is not an autonomous 𝛾-globin silencer, and thus by itself is not a suitable therapeutic target for gene editing treatment of ß-hemoglobinopathies.
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