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Article

Gene therapy for HbSC disease and other compound heterozygous sickle hemoglobinopathies: a time for inclusion.

Blood - 13 Nov 2025

Wilks Andrew, Steinberg Martin H, Frangoul Haydar

Abstract excerpt

ABSTRACT: Two gene therapy products have been approved by the US Food and Drug Administration for sickle cell disease. Nearly all patients in the clinical trials that led to approval either were sickle hemoglobin (HbS) gene homozygotes (sickle cell anemia) or had HbS-β0 thalassemia. HbSC disease, caused by compound heterozygosity for HbS and hemoglobin C genes, is the second most common genotype of sickle cell...

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Identifiers

PMID: 40834880
DOI: 10.1182/blood.2025029964

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