Lessons from Retinoblastoma: Implications for Cancer, Development, Evolution, and Regenerative Medicine

Trends Mol Med. 2016 Oct;22(10):863-876. doi: 10.1016/j.molmed.2016.07.010. Epub 2016 Aug 23.

Abstract

Retinoblastoma is a rare childhood cancer of the developing retina, and studies on this orphan disease have led to fundamental discoveries in cancer biology. Retinoblastoma has also emerged as a model for translational research for pediatric solid tumors, which is particularly important as personalized medicine expands in oncology. Research on retinoblastomas has been combined with the exploration of retinal development and retinal degeneration to advance a new model of cell type-specific disease susceptibility termed 'cellular pliancy'. The concept can even be extended to species-specific regeneration. This review discusses the remarkable path of retinoblastoma research and how it has shaped the most current efforts in basic, translational, and clinical research in oncology and beyond.

Keywords: cellular pliancy; orthotopic patient-derived xenografts; pediatric solid tumors; retinal regeneration; retinoblastoma.

Publication types

  • Review

MeSH terms

  • Animals
  • Child
  • Humans
  • Precision Medicine / methods
  • Regenerative Medicine / methods
  • Retina / growth & development
  • Retina / metabolism
  • Retina / pathology*
  • Retinal Neoplasms / genetics
  • Retinal Neoplasms / metabolism
  • Retinal Neoplasms / pathology*
  • Retinal Neoplasms / therapy
  • Retinoblastoma / genetics
  • Retinoblastoma / metabolism
  • Retinoblastoma / pathology*
  • Retinoblastoma / therapy
  • Translational Research, Biomedical / methods