Funding transnational collaborative research through joint transnational calls is one of the major objectives of E-Rare. This is the most important and effective joint activity to enhance the cooperation between European scientists working on rare diseases and thus reducing the fragmentation of research in this field. E-Rare launches calls on a yearly basis. The topic and eligibility criteria are specified every year and therefore may vary from one call to the other.

The Netherlands
Multi-omics approach to predict therapeutic targets for multiple system atrophy

Project Coordinator

Technical University of Munich


Otto Windl Ludwig-Maximilians-Universität München Munich, Germany
Jörg Tost Centre National de Recherche en Génomique Humain Evry , France
Hilal Lashuel School of Life Sciences Lausanne, Switzerland
Jeanine Houwing-Duistermaat University Medical Center Utrecht Utrecht, The Netherlands


Multiple system atrophy (MSA) is a devastating neurodegenerative disease, leading to death within 6-10 years. Symptoms of MSA affect the mobility and the autonomic nervous system. Available drugs only provide limited symptomatic relief. There is no curative therapy for MSA available. MSA is caused by aggregation of the protein alpha-synuclein in neurons and oligodendrocytes (support cells) in the brain. Deeper understanding of the molecular causes and consequences of alpha-synuclein aggregation, leading to neurodegeneration is highly important to develop better therapies. In preparatory work, we have already generated large datasets in patients-derived materials (genome-wide association study, epigenome-wide DNA methylation study) and corresponding cell models (DNA-methylome, miRnome, transcriptome, proteome, siRNA modifier screen, functional compound screen). In this project, we will  generate a large scale dataset on many biological levels (epigenome, transcriptome, proteome) of the two cell types affected in MSA (neurons, oligodendrocytes) derived from human post-mortem brains and induced pluripotent stem cells, from MSA patients and controls. These unique datasets will be explored by powerful computational methods to generate an integrated map of molecular pathways involved in MSA. This project is only possible in an international collaboration bringing together experts in various scientific areas. We expect to generate a unique and large database which will be shared with the scientific community to accelerate the development of novel breakthrough therapies for MSA.


E-Rare 2012 - Created by Toussaint Biger