In vivo models of brain tumors: roles of genetically engineered mouse models in understanding tumor biology and use in preclinical studies

Cell Mol Life Sci. 2014 Oct;71(20):4007-26. doi: 10.1007/s00018-014-1675-3. Epub 2014 Jul 10.


Although our knowledge of the biology of brain tumors has increased tremendously over the past decade, progress in treatment of these deadly diseases remains modest. Developing in vivo models that faithfully mirror human diseases is essential for the validation of new therapeutic approaches. Genetically engineered mouse models (GEMMs) provide elaborate temporally and genetically controlled systems to investigate the cellular origins of brain tumors and gene function in tumorigenesis. Furthermore, they can prove to be valuable tools for testing targeted therapies. In this review, we discuss GEMMs of brain tumors, focusing on gliomas and medulloblastomas. We describe how they provide critical insights into the molecular and cellular events involved in the initiation and maintenance of brain tumors, and illustrate their use in preclinical drug testing.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Brain Neoplasms / etiology*
  • Disease Models, Animal
  • Genetic Engineering*
  • Hedgehog Proteins / genetics
  • Hedgehog Proteins / metabolism
  • Proto-Oncogene Proteins c-myc / genetics
  • Proto-Oncogene Proteins c-myc / metabolism
  • Receptor, EphB2 / genetics
  • Receptor, EphB2 / metabolism
  • Tumor Suppressor Protein p53 / genetics
  • Tumor Suppressor Protein p53 / metabolism
  • Wnt Proteins / genetics
  • Wnt Proteins / metabolism


  • Hedgehog Proteins
  • Proto-Oncogene Proteins c-myc
  • Tumor Suppressor Protein p53
  • Wnt Proteins
  • Ephb2 protein, mouse
  • Receptor, EphB2