Back to search

Article

A prime editing strategy to rewrite the γ-globin promoters and reactivate fetal hemoglobin for sickle cell disease

2025-01-14

Abstract excerpt

Fetal hemoglobin (HbF) reactivation is a promising therapy for β-hemoglobinopathies. We developed a prime editing strategy that introduces multiple mutations in the fetal γ-globin promoters of patients’ hematopoietic stem/progenitor cells (HSPCs), boosting HbF expression and offering alternative therapeutic perspectives.

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work
b80a8cf1-ef79-55ec-8b83-0db2b363114f
DOI
10.1101/2025.01.13.632780
Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest
Interactive article relationship graphSelect a related publication card to move it into the centre and load its closest explainable connections. Solid lines are source-backed structured connections. Dashed lines are semantic discovery signals and are not scientific evidence.
A prime editing strategy to rewrite the γ-globin promoters and reactivate fetal hemoglobin for sickle cell diseaseDOI 10.1101/2025.01.13.632780
Enhanced γ-globin reactivation and sickle cell correction through a repressor-to-activator motif switch in the <i>HBG1/2</i> promotersSemantic proximity
Identification of novel HPFH-like mutations by CRISPR base editing that elevate the expression of fetal hemoglobinSemantic proximity
Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expressionSemantic proximity
A Small Key for a Heavy Door: Genetic Therapies for the Treatment of HemoglobinopathiesSemantic proximity
Identification of novel HPFH-like mutations by CRISPR base editing that elevates the expression of fetal hemoglobinSemantic proximity
Dissecting the epigenetic regulation of the fetal hemoglobin genes to unravel a novel therapeutic approach for β-hemoglobinopathiesSemantic proximity
Enhanced fetal hemoglobin production via dual-beneficial mutation editing of the HBG promoter in hematopoietic stem and progenitor cells for β-hemoglobinopathies.Semantic proximity
Editing the core region in HPFH deletions alters fetal and adult globin expression for treatment of β-hemoglobinopathiesSemantic proximity
Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34 <sup>+</sup> cell therapy to induce fetal hemoglobin for sickle cell diseaseSemantic proximity
Editing the core region in HPFH deletions alters fetal and adult globin expression for treatment of β-hemoglobinopathiesSemantic proximity
A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition.Semantic proximity
CRISPR-Cas9 to induce fetal hemoglobin for the treatment of sickle cell diseaseSemantic proximity
Select a neighboring publication to make it the new centre.