A next-generation dual-recombinase system for time- and host-specific targeting of pancreatic cancer

Nat Med. 2014 Nov;20(11):1340-1347. doi: 10.1038/nm.3646. Epub 2014 Oct 19.

Abstract

Genetically engineered mouse models (GEMMs) have dramatically improved our understanding of tumor evolution and therapeutic resistance. However, sequential genetic manipulation of gene expression and targeting of the host is almost impossible using conventional Cre-loxP-based models. We have developed an inducible dual-recombinase system by combining flippase-FRT (Flp-FRT) and Cre-loxP recombination technologies to improve GEMMs of pancreatic cancer. This enables investigation of multistep carcinogenesis, genetic manipulation of tumor subpopulations (such as cancer stem cells), selective targeting of the tumor microenvironment and genetic validation of therapeutic targets in autochthonous tumors on a genome-wide scale. As a proof of concept, we performed tumor cell-autonomous and nonautonomous targeting, recapitulated hallmarks of human multistep carcinogenesis, validated genetic therapy by 3-phosphoinositide-dependent protein kinase inactivation as well as cancer cell depletion and show that mast cells in the tumor microenvironment, which had been thought to be key oncogenic players, are dispensable for tumor formation.

Publication types

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

MeSH terms

  • Adenocarcinoma / metabolism
  • Adenocarcinoma / pathology
  • Animals
  • Carcinoma, Pancreatic Ductal / metabolism
  • Carcinoma, Pancreatic Ductal / pathology*
  • Cell Lineage
  • Female
  • Genetic Engineering / methods*
  • Green Fluorescent Proteins / metabolism
  • Male
  • Mast Cells / metabolism
  • Mast Cells / pathology
  • Mice
  • Models, Biological
  • Molecular Targeted Therapy*
  • Neoplasm Metastasis
  • Oncogenes
  • Pancreas / pathology
  • Precancerous Conditions / metabolism
  • Precancerous Conditions / pathology
  • Precision Medicine / methods*
  • Proto-Oncogene Proteins p21(ras) / metabolism
  • Recombinases / metabolism*
  • Reproducibility of Results
  • Species Specificity
  • Stromal Cells / metabolism
  • Stromal Cells / pathology
  • Tamoxifen
  • Time Factors

Substances

  • Recombinases
  • enhanced green fluorescent protein
  • Tamoxifen
  • Green Fluorescent Proteins
  • Hras protein, mouse
  • Proto-Oncogene Proteins p21(ras)