Science Translational Medicine
Science Translational Medicine
4 years ago

RNA binding proteins and the pathological cascade in ALS/FTD neurodegeneration.

Norihiro Suzuki, Mami Hatano, Daisuke Ito
Advanced genetic approaches have accelerated the identification of causative genes linked to the neurodegenerative diseases amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). Most of the disease-related proteins encoded by these genes form aggregates in the cellular machineries that regulate RNA and protein quality control in cells. Cross-talk among the signaling pathways governing these machineries leads to pathological cascades mediated by the accumulation of mutant RNA binding proteins. We outline the molecular basis of ALS and FTD pathogenesis and discuss the prospects for therapeutic strategies to treat these diseases.

Publisher URL: http://doi.org/10.1126/scitranslmed.aah5436

DOI: 10.1126/scitranslmed.aah5436

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