Gene therapy for hemoglobinopathies.

Authors

Nora M Gibson, Jaladhikumar Patel, Janet L Kwiatkowski

Published in

Seminars in hematology. Oct 01, 2026. Epub Oct 01, 2026.

Abstract

Gene modified hematopoietic stem cell transplantation represents a transformative treatment for hemoglobinopathies. Over 3 decades of study in the field have led to 2 commercial gene therapy products for transfusion-dependent thalassemia and sickle cell disease. Both commercial products demonstrate excellent efficacy in the short and intermediate term in improving the pathophysiology associated with these conditions. Numerous products are currently in development, which may further improve treatment outcomes. Challenges include optimizing stem cell collection in sickle cell disease, managing red cell and HLA alloimmunization, and preventing complications associated with busulfan conditioning. Further long-term studies are needed to better understand organ function, fertility, and risk of malignancy after gene therapy.

PMID:
43009425
Bibliographic data and abstract were imported from PubMed on 11 Nov 2026.

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