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CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
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Debating the Future of Sickle Cell Disease Curative Therapy: Haploidentical Hematopoietic Stem Cell Transplantation

Adetola A Kassim1, Alexis Leonard2,3

  • 1Department of Medicine, Division of Hematology/Oncology, Vanderbilt Meharry Sickle Cell Center of Excellence, Vanderbilt University School of Medicine, Nashville, TN 37232, USA.

Journal of Clinical Medicine
|August 26, 2022
PubMed

Hematopoietic stem cell transplantation (HSCT) is a well-established curative therapy for patients with sickle cell disease (SCD) when using a human leukocyte antigen (HLA)-matched sibling donor. Most patients with SCD do not have a matched sibling donor, thereby significantly limiting the accessibility of this curative option to most patients. HLA-haploidentical HSCT with post-transplant cyclophosphamide expands the donor pool, with current approaches now demonstrating high overall survival, reduced toxicity, and an effective reduction in acute and chronic graft-vs.-host disease (GvHD). Alternatively, autologous genetic therapies appear promising and have the potential to overcome significant barriers associated with allogeneic HSCT, such as donor availability and GvHD. Here the authors each take a viewpoint and discuss what will be the future of curative options for patients with SCD outside of a matched sibling transplantation, specifically haploidentical HSCT vs. gene therapy.

Keywords:
CRISPR/Cas9allogeneic transplantationautologous transplantationgene therapyhaploidenticalhematopoietic stem cell transplantationsickle cell disease

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