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Fetal hemoglobin induction via disruption of erythroid-specific enhancer elements (HbF Induction via Enhancer Disruption)
- Target
- HbF Induction via Enhancer Disruption
- Molecular classification
- Histone modification, Transcription factor regulation, Other
01
Overview
Fetal hemoglobin (HbF, α2γ2) induction is a therapeutic strategy aimed at treating β-hemoglobinopathies such as sickle cell disease and β-thalassemia by reactivating the expression of γ-globin genes in adult erythroid cells. This is achieved by disrupting erythroid-specific enhancer regions that regulate the expression of key transcriptional repressors (notably BCL11A) or directly modulate the chromatin architecture at the β-globin locus, leading to increased HbF production.
- Other names
- HBG reactivationFetal globin inductionγ-globin inductionBCL11A erythroid enhancer disruptionErythroid enhancer targeting for HbF induction
02
Mechanism of action
Disruption of erythroid-specific enhancers leads to derepression or reactivation of γ-globin gene expression, resulting in increased HbF production in adult red blood cells.
03
Biological functions
Gene expression regulationErythropoiesisHemoglobin switching
04
Disease associations
Sickle cell diseaseBeta-thalassemiaHemoglobinopathies
05
Safety considerations
Off-target effects of genome editingUnintended effects on erythropoiesisPotential for insertional mutagenesisImmune response to gene editing componentsLong-term effects of sustained HbF expression
06
Biomarkers
HbF levelsPercentage of HbF+ cellsBCL11A expression levels in erythroid cellsγ/β globin mRNA ratio
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