Targeting RNA-Binding proteins Roquin-1 and Regnase-1 could enhance CAR-iPSC-derived macrophage immunotherapy for solid tumors: a perspective and challenges

RNA Biol. 2025 Dec;22(1):1-7. doi: 10.1080/15476286.2025.2581385. Epub 2025 Oct 31.

Abstract

Solid tumours present major treatment obstacles because of their immunosuppressive microenvironment and poor response to traditional chimeric antigen receptor (CAR)-based immunotherapies. Recent advances in cellular engineering have introduced CAR-macrophages derived from induced pluripotent stem cells (CAR-iMacs) as a promising approach to get around these obstacles. CAR-iMacs are designed to attack tumours, but their phenotypic plasticity can cause them to transform into M2-like macrophages in the tumour environment (TME), where they may instead suppress immune responses and promote tumour progression and metastasis. Roquin-1 and Regnase-1 are RNA-binding proteins that act as negative regulators of inflammatory genes that contribute to the phenotypic plasticity of macrophages. This perspective highlights a novel approach to augmenting anti-tumour responses of CAR-iMacs by simultaneously knocking out Roquin-1 and Regnase-1 via CRISPR-Cas9 gene editing. This approach drives a shift from an immunosuppressive M2-like state to an M1 state, promoting sustained pro-inflammatory signalling, boosting phagocytic and cytotoxic capabilities within the tumour microenvironment. Addressing a serious constraint in conventional adoptive cell therapies, this dual-targeting platform could provide a potent and scalable immunotherapeutic treatment for solid malignancies.

Keywords: CAR-macrophages (CAR-iMacs); Regnase-1; Roquin-1; Solid tumours; immunotherapy.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • CRISPR-Cas Systems
  • Gene Editing
  • Humans
  • Immunotherapy* / methods
  • Immunotherapy, Adoptive* / methods
  • Induced Pluripotent Stem Cells* / cytology
  • Induced Pluripotent Stem Cells* / immunology
  • Induced Pluripotent Stem Cells* / metabolism
  • Macrophages* / immunology
  • Macrophages* / metabolism
  • Neoplasms* / genetics
  • Neoplasms* / immunology
  • Neoplasms* / therapy
  • RNA-Binding Proteins* / genetics
  • Receptors, Chimeric Antigen / genetics
  • Receptors, Chimeric Antigen / immunology
  • Receptors, Chimeric Antigen / metabolism
  • Ribonucleases* / genetics
  • Ribonucleases* / metabolism
  • Transcription Factors
  • Tumor Microenvironment / immunology
  • Ubiquitin-Protein Ligases* / genetics

Substances

  • Receptors, Chimeric Antigen
  • Ribonucleases
  • RNA-Binding Proteins
  • Ubiquitin-Protein Ligases
  • ZC3H12A protein, human
  • Transcription Factors