Generation and characterization of bioluminescent xenograft mouse models of MLL-related acute leukemias and in vivo evaluation of luciferase-targeting siRNA nanoparticles

Int J Oncol. 2012 Aug;41(2):621-8. doi: 10.3892/ijo.2012.1504. Epub 2012 May 31.

Abstract

Chromosomal translocations involving the MLL gene on 11q23 present frequent abnormalities in pediatric, adult and therapy-related acute leukemias, and are generally associated with aggressive disease and poor prognosis. Here, we report bioluminescent acute leukemia xenograft mouse models of the most frequent and aggressive MLL-related acute leukemias (infant and adult MLL-AF9, MLL-ENL, MLL-AF4). Four acute leukemia cell lines carrying MLL-related translocations were stably transduced with a firefly luciferase transgene and injected intravenously into NOD/SCID mice. Leukemia progression was monitored by in vivo bioluminescence imaging (BLI). All mice developed MLL-related acute leukemia. The four MLL-related acute leukemia models showed a different course of infant and adult MLL-AF9 acute myeloid leukemia, and a rapid aggressiveness of MLL-ENL acute lymphoblastic leukemia and MLL-AF4 acute biphenotypic leukemia. Tissue analysis and RT-PCR of bone marrow, spleen and liver from the mice confirmed the BL results. To validate BLI for the detection of a therapeutic response, systemic treatment with an anti-luciferase-targeting siRNA (siLuc) complexed with cationic nanoparticles was administered to mice with MLL-AF4 acute lymphoblastic leukemia. The BLI signal showed a reduction following treatment with siLuc compared to the control mice. These mouse models present MLL-related acute leukemia evolution similar to the human counterparts. Moreover, they are non-invasive, rapid and sensitive models, suitable for the in vivo study of MLL-related acute leukemias. Finally, BLI showed in vivo luminescence down modulation obtained by systemic treatment with luciferase-targeting siRNA nanoparticle complexes, confirming that these MLL-related leukemia mouse models are optimal for the evaluation and selection of delivery systems for siRNA and other new biotechnological pharmaceuticals.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Disease Models, Animal*
  • Disease Progression
  • Female
  • Gene Expression
  • Gene Knockdown Techniques
  • Histone-Lysine N-Methyltransferase
  • Humans
  • Leukemia, Biphenotypic, Acute / metabolism
  • Leukemia, Biphenotypic, Acute / pathology*
  • Liver / metabolism
  • Luciferases, Firefly / biosynthesis
  • Luciferases, Firefly / genetics*
  • Luminescent Measurements
  • Mice
  • Mice, Inbred NOD
  • Mice, SCID
  • Myeloid-Lymphoid Leukemia Protein / genetics
  • Nanoparticles / administration & dosage*
  • Neoplasm Transplantation
  • Oncogene Proteins, Fusion / genetics
  • RNA Interference
  • RNA, Small Interfering / genetics*
  • Spleen / metabolism
  • Spleen / pathology
  • Transfection
  • Whole Body Imaging

Substances

  • KMT2A protein, human
  • Oncogene Proteins, Fusion
  • RNA, Small Interfering
  • Myeloid-Lymphoid Leukemia Protein
  • Luciferases, Firefly
  • Histone-Lysine N-Methyltransferase