Abstract
The lysosome is a key catabolic organelle involved in many processes and its correct function is essential for health. The zebrafish tpp1 mutant is deficient in a key lysosome enzyme. It has a characteristic neurodegenerative phenotype, but with some variability in severity from animal to animal. In order to identify the gene(s) and protein(s) involved in this variability, we will look for genetic modifiers of the tpp1 mutant phenotype using a candidate pathway approach. The effect of one identified gene and its pathway will be characterized in detail in the zebrafish model to allow identification of potential pharmaceutical targets.
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