Parkinson’s Disease

Project open for codevelopment

Ksilink'project

Therapeutic area: CNS, neurodegenerative disorders

Disease: Parkinson’s Disease

Monogenic inroad: Alpha Synuclein (SNCA)

Cellular Model: Patient-derived cell lines

Project Origin: University of Luxembourg, Prof. Rejko Krüger

Medical need: Parkinson’s disease (PD) is the second most common neurodegenerative disorder currently affecting between 7-10 million people worldwide. The annual cost of PD in Europe is in excess of 14 billion euros (Olesen et al, Eur J Neurol, 2012). SNCA mutations are associated with autosomal dominant forms of PD. During the last decade it became more and moreclear that α-synuclein plays a central role in the pathogenesis of PD

GoalThe project aims for development and implementation of a physiologically highly-relevant cellular disease model based on differentiated induced pluripotent stem cells (iPSC), harbouring duplications and various mutations in SNCA, for the development of disease modifying treatments

Development stage: Assay validation

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