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000137108 0247_ $$2ISSN$$a1933-690X
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000137108 041__ $$aEnglish
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000137108 1001_ $$0P:(DE-HGF)0$$aMcCarthy, James M$$b0$$eCorresponding author
000137108 245__ $$aNanomedicine for prion disease treatment: new insights into the role of dendrimers.
000137108 260__ $$aLondon [u.a.]$$bTaylor & Francis$$c2013
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000137108 520__ $$aDespite their devastating impact, no effective therapeutic yet exists for prion diseases at the symptomatic stage in humans or animals. Progress is hampered by the difficulty in identifying compounds that affect PrP (Sc) and the necessity of any potential therapeutic to gain access to the CNS. Synthetic polymers known as dendrimers are a particularly promising candidate in this area. Studies with cell culture models of prion disease and prion infected brain homogenate have demonstrated that numerous species of dendrimers eliminate PrP (Sc) in a dose and time dependent fashion and specific glycodendrimers are capable of crossing the CNS. However, despite their potential a number of important questions remained unanswered such as what makes an effective dendrimer and how dendrimers eliminate prions intracellularly. In a number of recent studies we have tackled these questions and revealed for the first time that a specific dendrimer can inhibit the intracellular conversion of PrP (C) to PrP (Sc) and that a high density of surface reactive groups is a necessity for dendrimers in vitro anti-prion activity. Understanding how a therapeutic works is a vital component in maximising its activity and these studies therefore represent a significant development in the race to find effective treatments for prion diseases.
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000137108 650_7 $$2NLM Chemicals$$aDendrimers
000137108 650_7 $$2NLM Chemicals$$aPrions
000137108 650_2 $$2MeSH$$aAnimals
000137108 650_2 $$2MeSH$$aDendrimers: chemistry
000137108 650_2 $$2MeSH$$aDendrimers: pharmacology
000137108 650_2 $$2MeSH$$aDendrimers: therapeutic use
000137108 650_2 $$2MeSH$$aHumans
000137108 650_2 $$2MeSH$$aNanomedicine
000137108 650_2 $$2MeSH$$aPrion Diseases: drug therapy
000137108 650_2 $$2MeSH$$aPrion Diseases: metabolism
000137108 650_2 $$2MeSH$$aPrions: antagonists & inhibitors
000137108 650_2 $$2MeSH$$aPrions: chemistry
000137108 650_2 $$2MeSH$$aPrions: metabolism
000137108 650_2 $$2MeSH$$aProtein Conformation: drug effects
000137108 7001_ $$0P:(DE-HGF)0$$aAppelhans, Dietmar$$b1
000137108 7001_ $$0P:(DE-2719)9000396$$aTatzelt, Jörg$$b2$$udzne
000137108 7001_ $$0P:(DE-HGF)0$$aRogers, Mark S$$b3
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