From Prion Molecular Mechanisms to Targeted Therapeutics: Informing the Broader Proteinopathy Paradigm
A special issue of Pharmaceuticals (ISSN 1424-8247). This special issue belongs to the section "Pharmacology".
Deadline for manuscript submissions: 31 August 2026 | Viewed by 383
Editor
Interests: protein aggregation; neurodegeneration; prion diseases; proteinopathies; synucleinopathies; heparan sulfate proteoglycans; glycosaminoglycans
Special Issue Information
Dear Colleagues,
The contribution of prion researchers to science goes far beyond discovering a model for a rare group of neurodegenerative disorders. The prion field has been the canary in the coal mine for the neurodegenerative disease research community. Prion researchers provided the first definitive evidence that proteins alone self-propagate by converting endogenous protein into a pathogenic isoform that disrupts cellular homeostasis and drives progressive neurodegeneration. In recent decades, the concept of pathogenic proteins has been "rediscovered" or proven to apply to a wide range of proteinopathies, including the most common form of human dementia—Alzheimer’s disease—as well as Parkinson’s disease, amyotrophic lateral sclerosis, and frontotemporal dementia. The mechanistic similarities across proteinopathies gave rise to the controversial "prion-like" concept, which has been widely used by neuroscientists to refer to the propagation of pathologies, the seeding activity of proteins, and the characterization of protein strains.
Other lessons from the prion research field include the discovery of oligomeric protein aggregates as the primary neurotoxic forms in proteinopathies, the implementation of systems for the in vitro replication of pathogenic proteins to characterize strains and diagnose diseases, and the development of antibody-based therapies against the most toxic forms of protein aggregates. As prion researchers expand their interests to other proteinopathies, the opportunities to discover shared mechanisms of pathogenesis multiply, which is advancing the neurodegeneration research field significantly. In this review, we summarize the biggest breakthroughs in the prion research field that have contributed to the understanding of the pathogenic mechanisms and the development of therapeutic strategies across the spectrum of neurodegenerative proteinopathies.
Dr. Patricia Aguilar-Calvo
Guest Editor
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Keywords
- prion diseases
- prion-like
- protein aggregation
- proteinopathies
- neurodegeneration
- neurotoxicity
- diagnostics
- therapeutic strategies
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