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Allogeneic Transplant and Gene Therapy: Evolving Toward a Cure

R AdeBisi Lawal1, Mark C Walters2, Courtney D Fitzhugh3

  • 1Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institute, National Institutes of Health, 10 Center Drive, Building 10, Room 4-5140, Bethesda, MD 20892, USA; Hematology Branch, National Heart Lung and Blood Institute, National Institutes of Health, Bethesda, MD, USA.

Hematology/Oncology Clinics of North America
|November 18, 2022
PubMed

Curative therapies for sickle cell disease (SCD) include allogeneic human leukocyte antigen (HLA)- matched sibling and haploidentical hematopoietic cell transplant (HCT), gene therapy, and gene editing. However, comparative trial data that might facilitate selecting one curative therapy over another are unavailable. New strategies to decrease graft rejection and graft-versus-host disease (GVHD) risks are needed to expand haploidentical HCT. Myeloablative gene therapy and gene editing also has limitations. Herein, we review recent studies on curative therapies for SCD in the past 5 years.

Keywords:
Allogeneic transplantGene editingGene therapyGraft versus host diseaseHaploidenticalHematopoietic cell transplantMatched sibling donorSickle cell disease

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