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December 8, 2025BloodOpen Access

A genome-wide CRISPR activation screen identifies novel γ-globin regulators

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Authors

CMCarea MullinGMGreggory MyersZLZesen Lin

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Overview

Genome-wide pooled CRISPR activation screen reveals novel regulators enhancing hemoglobin expression, indicating new therapy potential for blood disorders.

Key Points

  • To identify novel regulators of γ-globin expression that may have therapeutic implications for β-hemoglobinopathies.
  • Performed a genome-wide pooled CRISPR activation screen in HUDEP-2 cells.
  • Generated lentiviral libraries and transduced cell lines to express sgRNAs targeting genes of interest.
  • Sorted and sequenced high and low γ-globin expressing cell populations for regulatory analysis.
  • Identified novel γ-globin activators such as HIC1, CHCHD7, FXR2, ANKH, and SKIL.
  • Activation of target genes led to a greater than 2-fold increase in γ-globin mRNA levels and %F-Cells in HUDEP-2 cells.
  • Validation in primary human erythroid cells confirmed a 3-fold increase in γ-globin expression with targeted gene activation.

Cite This Study

Mullin et al. (2025) studied this question.

synapsesocial.com/papers/69362f364fa91c937236d2f9https://doi.org/10.1182/blood-2025-1136
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Also Consider

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