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December 8, 2025Blood

Long-term follow-up of the first in human post-transcriptional genetic silencing of BCL11A in sickle cell disease in a phase 1 pilot and feasibility trial

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Authors

EEErica B. EsrickLLLeslie LehmannAFAmy Federico

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Overview

Pilot trial demonstrates gene therapy improved hemoglobin levels in sickle cell disease, indicating lasting effects and safety.

Key Points

  • Engrafted all 10 patients with sickle cell disease who received hematopoietic stem cells transduced with gene therapy.
  • Gene therapy led to a median HbF level increase to 27.8% by 2 years, stable at 48 months among 8 patients.
  • Observational analysis of clonal hematopoiesis found no significant abnormalities in gene sequencing after treatment.
  • Results highlight potential for gene therapy targeting BCL11A to improve sickle cell disease treatment outcomes.

Cite This Study

Esrick et al. (2025) studied this question.

synapsesocial.com/papers/69362f3a4fa91c937236d472https://doi.org/10.1182/blood-2025-2533
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Highly efficient collection and manufacture of autologous HSC gene therapy cell product for patients with sickle cell disease using a lentiviral vector containing a shmir targeting BCL11a2025 · 1 citations
  2. 2Evaluating the efficacy and safety of gene therapy in transfusion-dependent β-thalassemia: A focus on hemolysis improvement and clonal hematopoiesis monitoring2025 · 1 citations
  3. 3Targeted disruption of BCL11A ZnF4 enhances fetal hemoglobin in β-thalassemia and sickle cell disease: A druggable approach2025
  4. 4Real-world experience with apheresis for gene therapy in transfusion-dependent β-thalassemia: The largest single-center report2025
  5. 5CRISPR-Cas12a gene editing of the HBG1/2 promoters leads to sustained normalization of total hemoglobin and increased fetal hemoglobin in patients with severe sickle cell disease: Updated Results from the RUBY trial2025 · 2 citations