Understanding pRb: toward the necessary development of targeted treatments for retinoblastoma

J Clin Invest. 2012 Feb;122(2):425-34. doi: 10.1172/JCI57114. Epub 2012 Feb 1.


Retinoblastoma is a pediatric retinal tumor initiated by biallelic inactivation of the retinoblastoma gene (RB1). RB1 was the first identified tumor suppressor gene and has defined roles in the regulation of cell cycle progression, DNA replication, and terminal differentiation. However, despite the abundance of work demonstrating the molecular function and identifying binding partners of pRb, the challenge facing molecular biologists and clinical oncologists is how to integrate this vast body of molecular knowledge into the development of targeted therapies for treatment of retinoblastoma. We propose that a more thorough genetic understanding of retinoblastoma would inform targeted treatment decisions and could improve outcomes and quality of life in children affected by this disease.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Cell Cycle / physiology
  • Disease Models, Animal
  • Genes, Retinoblastoma
  • Genetic Therapy / methods
  • Humans
  • Mutation
  • Retinoblastoma / genetics
  • Retinoblastoma / pathology
  • Retinoblastoma / physiopathology
  • Retinoblastoma / therapy*
  • Retinoblastoma Protein / chemistry
  • Retinoblastoma Protein / genetics
  • Retinoblastoma Protein / metabolism*


  • Retinoblastoma Protein