The identification and characterization of putative disease-modifying pathways in neurodegenerative disorders has enormous potential for discovery of novel therapeutic agents that target these pathways. In studies of these putative pathways, it is critical to rigorously evaluate causality to the disease process. Findings in model organisms require validation in more physiologically relevant models but have the benefit of identifying targets amenable to modulation by small molecule therapeutics, possibly including drugs already in clinical use. Although many disease-modifying pathways have been reported, few have been validated in vivo in animal models of disease. Nonetheless, given the powerful genetic tools currently available, including a growing list of mouse mutants, the future looks promising and one can expect to see the number of clinical trials based on interventions that target disease-modifying pathways to continue growing.
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