Section 24
Chapter 23,601

Single treatment with RNAi against prion protein rescues early neuronal dysfunction and prolongs survival in mice with prion disease

White, M.D.; Farmer, M.; Mirabile, I.; Brandner, S.; Collinge, J.; Mallucci, G.R.

Proceedings of the National Academy of Sciences of the United States of America 105(29): 10238-10243


ISSN/ISBN: 0027-8424
PMID: 18632556
DOI: 10.1073/pnas.0802759105
Accession: 023600443

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Prion diseases are fatal neurodegenerative conditions for which there is no effective treatment. Prion propagation involves the conversion of cellular prion protein, PrP(C), to its conformational isomer, PrP(Sc), which accumulates in disease. Here, we show effective therapeutic knockdown of PrP(C) expression using RNAi in mice with established prion disease.

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