The TNF CNS Gene Therapy Group is interested in the molecular mechanisms of normal and pathological functions of neurons and myelin-forming cells in the central nervous system. Our main focus is on a group of neurological disorders termed leukodystrophies. The word leukodystrophy is of Greek origin and translates into ‘leuko = white’, ‘dys = abnormal’ and ‘troph = growth’. Leukodystrophies are genetic diseases of the brain white matter associated with an early onset and substantial mortality in children. The population incidence is estimated to be one in 7,600 live births and to date there are no effective treatments available. Our aim is to establish gene therapy protocols for leukodystrophies and related neurological disorders.
The first step in understanding these devastating conditions is to establish accurate, rodent disease models, which enable us to study the underlying pathophysiology and to explore novel therapeutic approaches. Once established, we leverage these models to develop novel gene therapy strategies. We have a long-standing interest in adeno-associated virus (AAV) as gene therapy vectors and as tools in basic research.
We are developing the current AAV platform in two directions:
Our research group employs state-of-the-art techniques, including behavioural testing, neurogenetics, molecular biology, histology and neuroimaging to characterise disease models and to assess the therapeutic outcome of gene therapy.
Dr Dominik Froehlich