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December 8, 2025Blood

Targeted disruption of BCL11A ZnF4 enhances fetal hemoglobin in β-thalassemia and sickle cell disease: A druggable approach

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Authors

SFSafana FarooqALAlshad S. LalaniOOOliver G. Ottmann

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Overview

Disruption of BCL11A ZnF4 enhances fetal hemoglobin in β-thalassemia and sickle cell disease, suggesting a potential gene editing therapy approach.

Key Points

  • Editing BCL11A ZnF4 led to increased fetal hemoglobin levels in patient-derived stem cells, indicating a potential therapeutic route.
  • Significant HbF expression increase (p < 0.001) was observed in edited hematopoietic stem cells and iPSCs with no impact on essential genes.
  • Using CRISPR-Cas9 for targeted gene editing in CD34+ cells yielded consistent results across different stem cell models.
  • The targeted delivery system effectively improved gene-editing uptake, highlighting its clinical potential in treating hemoglobinopathies.

Cite This Study

Farooq et al. (2025) studied this question.

synapsesocial.com/papers/69362f6e4fa91c937236e145https://doi.org/10.1182/blood-2025-6099
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Silencing of BCL11A by disrupting enhancer-dependent epigenetic insulation2025 · 1 citations
  2. 2Recapitulating HPFH by CRISPR-Cas9 editing of γ-globin regulators to reactivate γ-globin expression2025
  3. 3Multiplex base editing enhances fetal hemoglobin production in sickle cell disease erythroid cells2025
  4. 4Rapid, efficient and durable fetal hemoglobin production following CS-101 treatment in transfusion-dependent β-thalassemia participants: An autologous, ex vivo edited CD34+ stem cell product using the innovative transformer base editor (tBE)2025 · 3 citations
  5. 5Base editing of β 0 thalassemia mutations as a therapeutic strategy for β-hemoglobinopathies: efficacy and genotoxicity studies2025