Aebischer lab – Brain Mind Institute

Modelling and treatment of human neurodegenerative diseases in rodents.

Neurodegenerative disorders represent an heterogeneous group of disorders that usually strike in mid-life, causing progressive loss of motor and cognitive function. Although clinical manifestations vary, the outcome is the same: patients become incapacitated over a period of years and finally die. Tackling neurodegenerative diseases thus represents a formidable challenge for our ageing society.

A first axis of our research is focused on the development of rodent models to improve our understanding of the molecular basis of neurodegenerative diseases such as Parkinson’s disease (PD), Alzheimer’s disease (AD) and Motoneuron diseases (i.e. amyotrophic lateral sclerosis (ALS) and spino-muscular atrophy (SMA)).
On the other hand, we are developing gene therapy strategies aimed at correcting gene defects or treating its consequences, especially for gene deletion-based genetic disorders. To that purpose, we are using two distinct gene therapy approaches to deliver candidate genes in animal models of the diseases: (i) the encapsulation of genetically engineered cell lines releasing the therapeutic molecules (ex vivo gene therapy) and (ii) direct in vivo viral vector delivery (in vivo gene therapy).