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Advanced filters: Author: Elenoe C. Smith Clear advanced filters
  • A CRISPR-Cas9 approach is used to perform saturating mutagenesis of the human and mouse BCL11A enhancers, producing a map that reveals critical regions and specific vulnerabilities; BCL11A enhancer disruption is validated by CRISPR-Cas9 as a therapeutic strategy for inducing fetal haemoglobin by applying it in both mice and primary human erythroblast cells.

    • Matthew C. Canver
    • Elenoe C. Smith
    • Daniel E. Bauer
    Research
    Nature
    Volume: 527, P: 192-197