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Frangoul and colleagues: CRISPR–Cas9 editing for sickle-cell disease and β-thalassemia

Contribution grade

Clinical genetics · Early clinical intervention

Reference score145,000Provisional editorial estimate of this exact contribution; its grade is assessed separately.

What changed

Therapeutic genome editing showed early human feasibility in two monogenic blood diseases.

Scientific scope

Exact 26-author report and its two patients/follow-up; later cohorts and approval excluded.

Evidence

The linked sources identify this contribution and its scope. WRS has not independently reconstructed the scientific claims. The record is the exact 2021 NEJM report of two treated patients and its stated follow-up. The CRISPR mechanism paper, later trial cohorts, regulatory approvals, manufacturing and claims of universal cure remain separate.

Contributors

Provisional WRS assessment, edition 2026-10-16.1. Independent expert calibration is pending. This grade does not certify correctness or assign ownership.

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