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Review
. 2008 Jul;8(7):923-40.
doi: 10.1517/14712598.8.7.923.

Targeting prion proteins in neurodegenerative disease

Affiliations
Review

Targeting prion proteins in neurodegenerative disease

Sabine Gilch et al. Expert Opin Biol Ther. 2008 Jul.

Abstract

Background: Spongiform neurodegeneration is the pathological hallmark of individuals suffering from prion disease. These disorders, whose manifestation is sporadic, familial or acquired by infection, are caused by accumulation of the aberrantly folded isoform of the cellular prion protein (PrP(c)), termed PrP(Sc). Although usually rare, prion disorders are inevitably fatal and transferrable by infection.

Objective: Pathology is restricted to the central nervous system and premortem diagnosis is usually not possible. Yet, promising approaches towards developing therapeutic regimens have been made recently.

Methods: The biology of prion proteins and current models of neurotoxicity are discussed and prophylactic and therapeutic concepts are introduced.

Results/conclusions: Although various promising drug candidates with antiprion activity have been identified, this proof-of-concept cannot be transferred into translational medicine yet.

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