Disease-modifying pathways in neurodegeneration

Steven Finkbeiner, Ana Maria Cuervo, Richard I. Morimoto, Paul J. Muchowski

Research output: Contribution to journalArticlepeer-review

46 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)10349-10357
Number of pages9
JournalJournal of Neuroscience
Issue number41
StatePublished - Oct 11 2006

ASJC Scopus subject areas

  • Neuroscience(all)


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