PTBP1 and Cancer: From RNA Regulation to Therapeutic Potential

J Cell Mol Med. 2025 Jul;29(13):e70675. doi: 10.1111/jcmm.70675.

Abstract

Polypyrimidine tract binding protein 1 (PTBP1) belongs to the heterogeneous nuclear ribonucleoprotein (hnRNP) family and is a widely studied RNA-binding protein involved in regulating mRNA splicing, stability, localization, and translation. In addition, PTBP1 can participate in various non-coding RNA action processes and affect tumorigenesis and progression. In tumour therapy, PTBP1 may act as a key factor influencing targeted drug targets. Therefore, in this paper, we reviewed the structure and function of PTBP1, its role in various tumours, and key mechanisms of action. Moreover, we discuss the difficulties and challenges faced in PTBP1 research and the clinical translational potential of PTBP1 research and summarise the future research direction of PTBP1 to provide new ideas for basic research and new directions for precision treatment of tumours in the clinic.

Keywords: PTBP1; RNA modification; alternative splicing; targeted therapy; tumour translation.

Publication types

  • Review

MeSH terms

  • Animals
  • Gene Expression Regulation, Neoplastic
  • Heterogeneous-Nuclear Ribonucleoproteins* / chemistry
  • Heterogeneous-Nuclear Ribonucleoproteins* / genetics
  • Heterogeneous-Nuclear Ribonucleoproteins* / metabolism
  • Humans
  • Neoplasms* / genetics
  • Neoplasms* / metabolism
  • Neoplasms* / pathology
  • Neoplasms* / therapy
  • Polypyrimidine Tract-Binding Protein* / chemistry
  • Polypyrimidine Tract-Binding Protein* / genetics
  • Polypyrimidine Tract-Binding Protein* / metabolism

Substances

  • Polypyrimidine Tract-Binding Protein
  • Heterogeneous-Nuclear Ribonucleoproteins
  • PTBP1 protein, human