TNF CNS Gene Therapy

About us

image - Functional Genomics Group

from left to right: Georg von Jonquieres, Ziggy Harrison-Tikisci, Claudia Klugmann, Matthias Klugmann, Ben Rowlands, Dominik Fröhlich

We are generally interested in the molecular mechanisms of normal and pathological function of neurons and myelin forming cells in the central nervous system. Our aim is to establish gene therapy protocols for neurological disorders.

The success of our research relies on the availability of accurate animal models that we create by conventional transgenics or virus-mediated gene transfer. We have a long-standing interest in adeno-associated virus (AAV) as gene therapy vectors or as tools in basic research.

We are developing the current AAV platform in two directions:

(i) to achieve conditional transduction of specific neuronal sub-populations; and

(ii) to overcome the inherent neurotropism for targeting glia.

Using state-of-the-art techniques we employ a metabolomics approach, neuropharmacology, genetics, molecular biology and histology for our preclinical investigations on processes involved in memory formation and emotional behaviour but also severe conditions such as white matter disorders.

Please click on the link below to find more information on our current project:

A new strategy to treat myelin diseases





  • Caroline Rae, Neuroscience Research Australia (NeuRA), Australia
  • Beat Lutz, University Medical Center, University of Mainz, Germany
  • Miriam Schneider, Center for Mental Health, University of Heidelberg, Germany
  • Brad Alger, University of Maryland School of Medicine, Baltimore, Maryland, USA
  • Peter Kalivas, University of South Carolina, USA
  • Karen Szumlinski, University of Santa Barbara, USA
  • Sygnis Bioscience AG, Heidelberg, Germany
  • Giovanni Marsicano, INSERM Bordeaux, France

Grants & Funding

  • National Health & Medical Research Council (NHMRC) Project grant APP1050277 “Non-invasive gene delivery for expression of therapeutic genes in oligodendrocytes: A new strategy to treat myelin diseases.”
  • Mission Massimo Foundation Project grant: “Homing in on modeling and treating tRNA synthetase disorders.”
  • Gandel Philanthropy Project grant: “Gene therapy for DARS - a new childhood leuodystrophy.”
  • University of New South Wales (UNSW) Gold Star – NHMRC (Collaborative Grant with Prof. Caroline Rae): “Control of Brain Metabolism and activity by acetylation.”
  • Australian Research Council


  • Ziggy Harrison-Tikisci (PhD)
  • Benjamin Rowlands (PhD)