Claudio Soto1, Zane Martin. 1. University of Texas Medical Branch, George and Cynthia Mitchell Center for Neurodegenerative Diseases, Departments of Neurology, Neuroscience and Cell Biology and Biochemistry and Molecular Biology, 301 University Blvd, TX 77555, Galveston, USA +1 409 7470017 ; +1 409 7470020 ; clsoto@utmb.edu.
Abstract
BACKGROUND: Neurodegenerative diseases are a group of chronic and progressive disorders of the nervous system. A hallmark event in these diseases is the misfolding and accumulation in the brain of protein aggregates. OBJECTIVE: In this article, we describe the knowledge of the mechanism of protein misfolding and aggregation, its role in neurodegeneration and the diverse therapeutic targets for intervention. We also critically review various strategies under development to discover drugs attacking this process. CONCLUSION: In spite of the substantial progress on understanding the critical role of protein misfolding and aggregation, drugs effective against this process are still years away from approval.
BACKGROUND:Neurodegenerative diseases are a group of chronic and progressive disorders of the nervous system. A hallmark event in these diseases is the misfolding and accumulation in the brain of protein aggregates. OBJECTIVE: In this article, we describe the knowledge of the mechanism of protein misfolding and aggregation, its role in neurodegeneration and the diverse therapeutic targets for intervention. We also critically review various strategies under development to discover drugs attacking this process. CONCLUSION: In spite of the substantial progress on understanding the critical role of protein misfolding and aggregation, drugs effective against this process are still years away from approval.