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Showing 1–1 of 1 articles · Keyword: Sickle cell disease Clear keyword

Seminars in hematology2026ResearchNon-viral Gene Delivery

1. Sickle cell disease-specific barriers to in vivo hematopoietic stem cell gene therapy

Choi Rh, Sheehan Va

Gene therapy for individuals living with sickle cell disease is a transformative therapy, ameliorating many of its severe symptoms. Nevertheless, ex vivo administration of gene therapy coupled with sickle cell disease specific obstacles, complicate and limit widespread adoption. In this review, we discuss current…

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