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Review

Therapeutic approaches for prion disorders

, , , , &
Pages 613-630 | Published online: 10 Jan 2014
 

Abstract

Prion diseases are lethal for both humans and animals, and affected individuals die after several months following a rapid disease progression. Although researchers have attempted for decades to develop effective therapeutics for the therapy of human prion disorders, until now no efficient drug has been available on the market for transmissible spongiform encephalopathy (TSE) treatment or cure. Approximately 200 patients worldwide have died or suffer from variant Creutzfeldt–Jakob disease (CJD). Incidences for sporadic and familial CJD are approximately 1.5–2 per million per year and one per 10 million per year, respectively, in Europe. This review summarizes classical and modern trials for the development of effective anti-TSE drugs, introduces potential effective delivery systems, such as lentiviral and adeno-associated virus systems for antiprion components, including antibodies and siRNAs, and presents vaccination trials. Most of the antiprion drugs target prion protein PrPc and/or PrPSc. Alternative targets are receptors and coreceptors for PrP, that is, the 37/67-kDa laminin receptor and heparan sulfate proteoglycanes. We review clinical trials for the treatment of TSEs and describe hindrances and chances for a breakthrough in therapy of prion disorders.

Acknowledgements

This work was supported by the European Commission (grant NoE-NeuroPrion FOOD-CT-2004–506579, the Bundeministerium für Bildung und Forschung (grant 01-KO-0514) and the Deutsche Forschungsgemeinschaft (grant WE 2664/2–1).

Financial disclosure

The authors have no relevant financial interests related to this manuscript, including employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

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