Positron emission tomography reporter gene strategy for use in the central nervous system

Proc Natl Acad Sci U S A. 2019 Jun 4;116(23):11402-11407. doi: 10.1073/pnas.1901645116. Epub 2019 May 23.

Abstract

There is a growing need for monitoring or imaging gene therapy in the central nervous system (CNS). This can be achieved with a positron emission tomography (PET) reporter gene strategy. Here we report the development of a PET reporter gene system using the PKM2 gene with its associated radiotracer [18F]DASA-23. The PKM2 reporter gene was delivered to the brains of mice by adeno-associated virus (AAV9) via stereotactic injection. Serial PET imaging was carried out over 8 wk to assess PKM2 expression. After 8 wk, the brains were excised for further mRNA and protein analysis. PET imaging at 8 wk post-AAV delivery showed an increase in [18F]DASA-23 brain uptake in the transduced site of mice injected with the AAV mice over all controls. We believe PKM2 shows great promise as a PET reporter gene and to date is the only example that can be used in all areas of the CNS without breaking the blood-brain barrier, to monitor gene and cell therapy.

Keywords: PKM2; [18F]DASA-23; adeno-associated virus; positron emission tomography (PET); reporter gene.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Cell Line, Tumor
  • Central Nervous System / metabolism*
  • Central Nervous System / virology
  • Dependovirus / genetics
  • Female
  • Fluorine Radioisotopes / administration & dosage
  • Genes, Reporter / genetics*
  • Genetic Therapy / methods
  • HeLa Cells
  • Humans
  • Mice
  • Mice, Inbred BALB C
  • Positron-Emission Tomography / methods

Substances

  • Fluorine Radioisotopes