Protein folding and misfolding neurodegenerative diseases focus on structural biology volume 7 series editor rob kaptein bijvoet center for biomolecular research utrecht university the netherlands. This video is unavailable watch queue queue watch queue queue. The first known protein misfolding disease indeed the first inherited human disease to have a known molecular mechanism was sickle cell anemia a related example of this category of protein folding diseases is provided by gauchers disease the most common lysosomal storage disease neurodegenerative disease amyloid pores from . A hallmark of neurodegenerative proteinopathies is the formation of misfolded protein aggregates that cause cellular toxicity and contribute to cellular proteostatic collapse therapeutic options are currently being explored that target different steps in the production and processing of proteins implicated in neurodegenerative disease including synthesis chaperone assisted folding and
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