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Posted: 2025-04-23 06:58:57 UTC
This article contains some claims that remain unverified. While much of the content may be accurate, exercise care when relying on this information.
This article contains some claims that remain unverified. While much of the content may be accurate, exercise care when relying on this information.
Status
Last Updated
2025-04-23 06:59:13 UTC
Verified By
Rollup News
This pilot trial investigates modified lentiviral globin gene therapy for pediatric patients with β0/β0 transfusion-dependent β-thalassemia, conducted at a single center.
Gene therapy for β-thalassemia
Pediatric hematology
Lentiviral vector modification
Transfusion independence
Transfusion dependence in β-thalassemia patients
Efficacy of gene therapy
Long-term outcomes