Non-genotoxic conditioning for hematopoietic stem cell transplantation using a hematopoietic-cell-specific internalizing immunotoxin

Nat Biotechnol. 2016 Jul;34(7):738-45. doi: 10.1038/nbt.3584. Epub 2016 Jun 6.

Abstract

Hematopoietic stem cell transplantation (HSCT) offers curative therapy for patients with hemoglobinopathies, congenital immunodeficiencies, and other conditions, possibly including AIDS. Autologous HSCT using genetically corrected cells would avoid the risk of graft-versus-host disease (GVHD), but the genotoxicity of conditioning remains a substantial barrier to the development of this approach. Here we report an internalizing immunotoxin targeting the hematopoietic-cell-restricted CD45 receptor that effectively conditions immunocompetent mice. A single dose of the immunotoxin, CD45-saporin (SAP), enabled efficient (>90%) engraftment of donor cells and full correction of a sickle-cell anemia model. In contrast to irradiation, CD45-SAP completely avoided neutropenia and anemia, spared bone marrow and thymic niches, enabling rapid recovery of T and B cells, preserved anti-fungal immunity, and had minimal overall toxicity. This non-genotoxic conditioning method may provide an attractive alternative to current conditioning regimens for HSCT in the treatment of non-malignant blood diseases.

MeSH terms

  • Animals
  • Antibodies, Monoclonal / genetics
  • Antibodies, Monoclonal / immunology
  • Cell Differentiation / genetics
  • Cell Differentiation / immunology
  • Cell Survival / genetics
  • Cell Survival / immunology
  • Cells, Cultured
  • DNA Damage / genetics
  • DNA Damage / immunology*
  • Female
  • Genetic Enhancement / methods
  • Hematopoietic Stem Cell Transplantation / methods*
  • Hematopoietic Stem Cells / immunology*
  • Immunogenetic Phenomena / genetics
  • Immunotoxins
  • Leukocyte Common Antigens / immunology*
  • Mice
  • Mice, Inbred C57BL
  • Ribosome Inactivating Proteins, Type 1 / genetics*
  • Ribosome Inactivating Proteins, Type 1 / immunology*
  • Saporins

Substances

  • Antibodies, Monoclonal
  • Immunotoxins
  • Ribosome Inactivating Proteins, Type 1
  • Leukocyte Common Antigens
  • Ptprc protein, mouse
  • Saporins