Antigen Presentation by Individually Transferred HLA Class I Genes in HLA-A, HLA-B, HLA-C Null Human Cell Line Generated Using the Multiplex CRISPR-Cas9 System

J Immunother. 2017 Jul/Aug;40(6):201-210. doi: 10.1097/CJI.0000000000000176.

Abstract

Human leukocyte antigens (HLAs) are essential immune molecules that affect transplantation and adoptive immunotherapy. When hematopoietic stem cells or organs are transplanted with HLA-mismatched recipients, graft-versus-host disease or graft rejection can be induced by allogeneic immune responses. The function of each HLA allele has been studied using HLA-deficient cells generated from mutant cell lines or by RNA interference, zinc finger nuclease, and the CRISPR/Cas9 system. To improve HLA gene editing, we attempted to generate an HLA class I null cell line using the multiplex CRISPR/Cas9 system by targeting exons 2 and 3 of HLA-A, HLA-B, and HLA-C genes simultaneously. Multiplex HLA editing could induce the complete elimination of HLA class I genes by bi-allelic gene disruption on target sites which was defined by flow cytometry and target-specific polymerase chain reaction. Furthermore, artificial antigen-presenting cells were generated by transfer of a single HLA class I allele and co-stimulatory molecules into this novel HLA class I null cell line. Artificial antigen-presenting cells showed HLA-restricted antigen presentation following antigen processing and were successfully used for the efficient generation of tumor antigen-specific cytotoxic T cells in vitro. The efficient editing of HLA genes may provide a basis for universal cellular therapies and transplantation.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antigen-Presenting Cells / physiology*
  • Antigen-Presenting Cells / transplantation
  • Antigens, Neoplasm / immunology
  • Antigens, Neoplasm / metabolism
  • CRISPR-Cas Systems*
  • Cell Line
  • Exons / genetics
  • Gene Editing
  • Graft Rejection / immunology
  • Graft Rejection / prevention & control*
  • Graft vs Host Disease / immunology
  • Graft vs Host Disease / prevention & control*
  • HLA-A Antigens / genetics*
  • HLA-A Antigens / metabolism
  • HLA-B Antigens / genetics*
  • HLA-B Antigens / metabolism
  • HLA-C Antigens / genetics*
  • HLA-C Antigens / metabolism
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Immunotherapy, Adoptive / methods*
  • Lymphocyte Activation
  • T-Lymphocytes, Cytotoxic / immunology*
  • T-Lymphocytes, Cytotoxic / transplantation

Substances

  • Antigens, Neoplasm
  • HLA-A Antigens
  • HLA-B Antigens
  • HLA-C Antigens