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To pursue gene therapy or not? Is it feasible after graft failure in allogeneic hematopoietic cell transplant recipients

5 Jun 2025

Abstract excerpt

ABSTRACT: Two autologous hematopoietic stem cell (HSC)-based gene therapies (GTs) are now commercially available for severe sickle cell disease and transfusion-dependent β-thalassemia. However, the safety and efficacy of a subsequent autologous HSC-based GT after graft failure with a previous allogeneic hematopoietic cell transplant (HCT) remains unclear. Some individuals who have experienced a failed first...

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PMID: 40472294
DOI: 10.1182/bloodadvances.2024015413

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