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August 20, 2025Open Access

Recapitulating HPFH by CRISPR-Cas9 editing of γ-globin regulators to reactivate γ-globin expression

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Authors

YLYan LiMWMingyue WangQLQingyuan Luo

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Overview

Genome editing reactivates fetal hemoglobin in patients with β-hemoglobinopathies, suggesting a potential new therapy.

Key Points

  • This approach achieved robust fetal hemoglobin reactivation in various experimental systems, including K562 cells.
  • The study utilized a dual-editing strategy involving BCL11A disruption and HPFH-associated deletion recapitulation.
  • The CRISPR-Cas9 method shows promise as a transformative treatment for β-thalassemia and sickle cell disease.
  • Optimized delivery of Cas9: sgRNA ribonucleoprotein significantly enhanced gene editing efficiency and efficacy.

Cite This Study

Li et al. (2025) studied this question.

synapsesocial.com/papers/68af4eb4ad7bf08b1ead75ffhttps://doi.org/10.21203/rs.3.rs-7301640/v1
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Also Consider

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

  1. 1Multiplex CRISPR/Cas9 genome editing in hematopoietic stem cells for fetal hemoglobin reinduction generates chromosomal translocations2021 · 51 citations
  2. 2Targeted disruption of BCL11A ZnF4 enhances fetal hemoglobin in β-thalassemia and sickle cell disease: A druggable approach2025
  3. 3Epigenomic Reactivation of Fetal Hemoglobin via BCL11A Enhancer Editing A Proposed Durable Strategy for Malaria Prevention2026
  4. 4A genome-wide CRISPR activation screen identifies novel γ-globin regulators2025
  5. 5Optimized CRISPR-Cas12a genome-wide screen reveals PTPA phosphatase pathway in fetal hemoglobin silencing2026 · 2 citations