Allogeneic Transplant and Gene Therapy: Evolving Toward a Cure

Hematol Oncol Clin North Am. 2022 Dec;36(6):1313-1335. doi: 10.1016/j.hoc.2022.06.007.

Abstract

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 transplant; Gene editing; Gene therapy; Graft versus host disease; Haploidentical; Hematopoietic cell transplant; Matched sibling donor; Sickle cell disease.

Publication types

  • Review
  • Research Support, N.I.H., Extramural
  • Research Support, N.I.H., Intramural

MeSH terms

  • Allografts
  • Anemia, Sickle Cell* / genetics
  • Anemia, Sickle Cell* / therapy
  • Genetic Therapy
  • Graft vs Host Disease* / etiology
  • Graft vs Host Disease* / prevention & control
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Transplantation Conditioning