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

Prion diseases are fatal neurodegenerative conditions for which there is no effective treatment. Prion propagation involves the conversion of cellular prion protein, PrPC, to its conformational isomer, PrPSc, which accumulates in disease. Here, we show effective therapeutic knockdown of PrPC express...

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Veröffentlicht in:	Proceedings of the National Academy of Sciences - PNAS 2008-07, Vol.105 (29), p.10238-10243
Hauptverfasser:	White, Melanie D, Farmer, Michael, Mirabile, Ilaria, Brandner, Sebastian, Collinge, John, Mallucci, Giovanna R
Format:	Artikel
Sprache:	eng
Schlagworte:	Animals Base Sequence Behavior, Animal Biological Sciences Burrowing Genes, Reporter Genetic Therapy - methods Green Fluorescent Proteins - genetics Hippocampus Hippocampus - pathology Hippocampus - physiopathology Infections Lentivirus Lentivirus - genetics Medical treatment Mice Mice, Transgenic Molecular Sequence Data Neurological disorders Neurons Prion diseases Prion Diseases - genetics Prion Diseases - physiopathology Prion Diseases - psychology Prion Diseases - therapy Prion Proteins Prions Prions - antagonists & inhibitors Prions - genetics Prions - metabolism Proteins PrPC Proteins - antagonists & inhibitors PrPC Proteins - genetics PrPC Proteins - metabolism PrPSc Proteins - metabolism Ribonucleic acid RNA RNA Interference RNA, Messenger - genetics RNA, Messenger - metabolism RNA, Small Interfering - administration & dosage RNA, Small Interfering - genetics Rodents T tests Viruses
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