TREATPOLYQ · TreatPolyQ – Industrial Academic Initial Training Network towards treatment of Polyglutamine Diseases

Protein aggregation is a hallmark of many late onset neurodegenerative disorders including Parkinson’s Disease (PD), Alzheimer’s Disease (AD), amyotrophic lateral sclerosis (ALS), prion diseases as well as the group of polyglutamine diseases (polyQ). The aim of this proposal is to create a network of European partners bridging important basic mechanisms involved in proteinopathies, research of model diseases and treatment approaches. The “TreatPolyQ” network will focus on two main representatives of the polyQ diseases: Huntington’s disease as the most common polyQ disease as well as spinocerebellar ataxia type 3 (SCA3) as the most frequent autosomal-dominantly inherited ataxia. Patients suffer from a multitude of neurological symptoms including movement abnormalities with late onset and in a progressive manner. Up to now, no treatment or cure is available. The network will be consisting of a rare combination of experts from basic and translational research, including a Nobel prize laureate, four industrial partners (two medium, and two small companies, all incorporated as full participants) and academic leaders of the field. The network not only focuses on one special aspect of a disease but spans several important disease-associated mechanism as well as promising treatment strategies for HD and SCA3 (protein transport, protein folding, protein degradation via both the ubiquitin-proteasome system and autophagy), likely to be important across a range of neurodegenerative diseases. In order to implement these research projects, extensive collaborations and temporarily personnel secondments of the involved researchers will take place, enhancing interdisciplinary transfer of knowledge. Beyond the personalized local training plan for each employed researcher within the Network, there will be 4 structured courses covering aspects ranging from structural biology to protein degradation to model organisms and drug development, including soft skill training.