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Taylor and Francis Group, Prion, 3(3), p. 121-128

DOI: 10.4161/pri.3.3.9289

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Therapy for prion diseases: Insights from the use of RNA interference

Journal article published in 2009 by White Md, Melanie D. White ORCID, Mallucci Gr, Giovanna R. Mallucci ORCID
This paper is available in a repository.
This paper is available in a repository.

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Abstract

Insights into the molecular basis and the temporal evolution of neurotoxicity in prion disease are increasing, and recent work in mice leads to new avenues for targeting treatment of these disorders. Using lentivirally mediated RNA interference (RNAi) against native prion protein (PrP), White et al. report the first therapeutic intervention that results in neuronal rescue, prevents symptoms and increases survival in mice with established prion disease.(1) Both the target and the timing of treatment here are crucial to the effectiveness of this strategy: the formation of the neurotoxic prion agent is prevented at a point when diseased neurons can still be saved from death. But the data also give new insights into the timing of treatment in the context of the pattern of spread of prion infection throughout the brain, with implications for developing the most effective treatments.