Gene therapy using haematopoietic stem and progenitor cells

Nat Rev Genet. 2021 Apr;22(4):216-234. doi: 10.1038/s41576-020-00298-5. Epub 2020 Dec 10.

Abstract

Haematopoietic stem and progenitor cell (HSPC) gene therapy has emerged as an effective treatment modality for monogenic disorders of the blood system such as primary immunodeficiencies and β-thalassaemia. Medicinal products based on autologous HSPCs corrected using lentiviral and gammaretroviral vectors have now been approved for clinical use, and the site-specific genome modification of HSPCs using gene editing techniques such as CRISPR-Cas9 has shown great clinical promise. Preclinical studies have shown engineered HSPCs could also be used to cross-correct non-haematopoietic cells in neurodegenerative metabolic diseases. Here, we review the most recent advances in HSPC gene therapy and discuss emerging strategies for using HSPC gene therapy for a range of diseases.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • CRISPR-Cas Systems / genetics
  • Gene Editing / methods
  • Genetic Therapy*
  • Hematopoietic Stem Cell Transplantation / methods*
  • Hematopoietic Stem Cells / cytology
  • Humans
  • Primary Immunodeficiency Diseases / genetics
  • Primary Immunodeficiency Diseases / therapy*
  • beta-Thalassemia / genetics
  • beta-Thalassemia / therapy*