Exosome-based nanomedicine for the treatment of white matter diseases

• Applicant: Dr. Dominik Fröhlich
• Subject Area: Molecular and Cellular Neurology and Neuropathology ; Molecular Biology and Physiology of Neurons and Glial Cells
• Term: from 2014 to 2016
• Project identifier: Deutsche Forschungsgemeinschaft (DFG) - Project number 259937163

This project aims at exploiting exosomes as a delivery platform for the treatment of white matter diseases. Exosomes are cell-derived extracellular microvesicles that relay information in form of proteins or nucleic acids between cells. By the expression of targeting ligands exosomes hold enormous potential for directed transfer of therapeutic agents in nanomedicine applications. Oligodendrocytes are the myelinating cells of the central nervous system. Apart from implementing fast saltatory impulse conduction by insulation of axons with a myelin sheath, they provide trophic support to neurons essential for long-term axonal integrity. Single gene mutations causing oligodendrocyte dysfunction underlie leukodystrophies, inherited myelin diseases associated with substantial morbidity and mortality in children. Gene therapy has emerged as a promising experimental treatment strategy for leukodystrophies but success has been hampered by the lack of drug delivery systems that target oligodendrocytes. In this project, we will identify recombinant exosomes for targeted delivery of either therapeutic protein or the corresponding cDNA to restore function of oligodendrocytes in an accurate mouse model of the leukodystrophy Canavan disease. If successful, this research will create novel treatment options not only for white matter diseases but whenever safe and efficient delivery of molecules to oligodendrocytes is required.

• DFG Programme: Research Fellowships
• International Connection: Australia
• Host: Professor Dr. Matthias Klugmann