Research
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Genome editing of HBG1 and HBG2 to induce fetal hemoglobin.
Abstract
Induction of fetal hemoglobin (HbF) via clustered regularly interspaced short palindromic repeats/Cas9-mediated disruption of DNA regulatory elements that repress γ-globin gene (HBG1 and HBG2) expression is a promising therapeutic strat...
Publication · November 02, 2025
Chromothripsis as an on-target consequence of CRISPR-Cas9 genome editing
Abstract
Genome editing has therapeutic potential for treating genetic diseases and cancer. However, the currently most practicable approaches rely on the generation of DNA double-strand breaks (DSBs), which can give rise to a poorly characteriz...
Publication · November 02, 2025
Activation of γ-globin gene expression by GATA1 and NF-Y in hereditary persistence of fetal hemoglobin
Abstract
Hereditary persistence of fetal hemoglobin (HPFH) ameliorates β-hemoglobinopathies by inhibiting the developmental switch from γ-globin (HBG1/HBG2) to β-globin (HBB) gene expression. Some forms of HPFH are associated with γ-globin promo...
Publication · November 02, 2025
Genetic therapies for the first molecular disease.
Abstract
Sickle cell disease (SCD) is a monogenic disorder characterized by recurrent episodes of severe bone pain, multi-organ failure, and early mortality. Although medical progress over the past several decades has improved clinical outcomes ...
Publication · November 02, 2025
Limitations of mouse models for sickle cell disease conferred by their human globin transgene configurations
Abstract
ABSTRACT 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 ...
Publication · November 02, 2025
Dual function NFI factors control fetal hemoglobin silencing in adult erythroid cells
Publication · November 02, 2025
Potent and uniform fetal hemoglobin induction via base editing
Abstract
A comparison of fetal hemoglobin gene editing strategies using human sickle cell disease donor cells and in vivo transplantation finds that adenine base editing of the –175A>G site in the γ-globin gene promoters results in durable and p...
Publication · November 02, 2025
Hydroxyurea reduces the levels of the fetal globin gene repressors ZBTB7A/LRF and BCL11A in erythroid cells in vitro
Abstract
Abstract Objectives Hydroxyurea (HU) is the most widely used therapy for adults and children with sickle cell disease (SCD). It is believed to act largely by inducing the transcription of fetal γ-globin genes to generate fetal hemoglobi...
Publication · November 02, 2025
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