X-linked Adrenoleukodystrophy (X-ALD), the most common peroxisomal disorder, is an inherited neurodegenerative disorder that is associated with inflammation and irreversible destruction of myelin in the brain. We are using cell culture and mouse models that lack functional ALD protein to study the basic pathomechanisms of X-ALD and to test novel molecular strategies for therapeutic intervention (pharmacological gene therapy).
- an overview of our understanding of X-ALD -more-
- generation and use of mouse models for X-ALD
- molecular basis for clinical heterogeneity in X-ALD -more-
- the role of mitochondria in X-ALD -more-
- expression and function of ALD-protein -more-
- pharmacological gene therapy in X-ALD -more-
Functions of Peroxisomes in the Central Nervous System
We are interested in the proteom of peroxisomes in different tissues, in order to elucidate novel peroxisomal pathways and functions important for the CNS. In addition, we have generated a transgenic mouse model with green fluorescent peroxisomes which, together with other mouse models are used to explain specific questions concerning the peroxisomes and the nervous system.
- proteom of peroxisomes -more-
- generation of mouse models to study peroxisomes in the CNS -more-
- the role of peroxisomes in cholesterol synthesis -more-
- peroxisomes as modulator or modifier in common neurodegenerative disorders
- Parkinson's Disease -more-
- Alzheimer's Disease -more-
Metachromatic leukodystrophy is an inherited sulfatide storage disease, classically due to deficiency of the lysosomal enzyme arylsulfatase A. We have been interested in the genotype-phenotype relation in this leukodystrophy -more-