A hallmark of neurodegenerative proteinopathies is the formation of misfolded protein aggregates, so therapeutic options are currently being explored to target different steps in the production and processing of proteins implicated in neurodegenerative disease. However, there are major challenges that impact the development of novel therapies, including incomplete knowledge of disease targets and a lack of biomarkers to monitor disease progression and therapeutic response.

This review paper is partly based on presentations at “Protein Misfolding – Implications and Strategies,” an open scientific satellite symposium held in conjunction with the Society for Neuroscience’s 45th Annual Meeting in 2015, sponsored and organized by Charles River Laboratories. The panelists included several authors of the review paper.