Approaches of research

Following approaches of research and cure can be basically differentiated:

• Works on model-organisms to enlighten the mechanisms of disease. Therefore, e.g. nematodes which have a well characterized nervous system are suitable. Prof. Baumeister is a leading scientist in this field. Furthermore yeast has a gene, close related to the human CLN3. This is the focus of Prof. Pearce´s group. Corresponding mouse models are investigated in the newly built EU-project "NCL-models".
• Experimental use of medicaments: There are a number of medicaments, which achieve promising success in comparable diseases, for example CPHPC, Flupirtin or Cystagon. These medicaments were applied either in diseases with similar symptoms or in cell-cultures of NCL-patients. But so far it only could be demonstrated that the biochemical appearances were attenuated.
• Substitution of the missing protein: In the juvenile NCL the protein CLN3, which is a lysosomal membrane protein, is missing. The defect is directly associated with a chloride-channel, which is responsible for the degradation of harmful substances, generated by metabolism. One of the German experts in this field is Prof. Sandhoff.
• Gene-therapy: In the end, to antagonize the real cause it is essential to eliminate the gene-defect on chromosome 16. Only then it is ensured, that not every cell is affected because of cell division processes.