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@ARTICLE{McCarthy:137108,
author = {McCarthy, James M and Appelhans, Dietmar and Tatzelt, Jörg
and Rogers, Mark S},
title = {{N}anomedicine for prion disease treatment: new insights
into the role of dendrimers.},
journal = {Prion},
volume = {7},
number = {3},
issn = {1933-6896},
address = {London [u.a.]},
publisher = {Taylor $\&$ Francis},
reportid = {DZNE-2020-03430},
pages = {198-202},
year = {2013},
abstract = {Despite 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.},
keywords = {Animals / Dendrimers: chemistry / Dendrimers: pharmacology
/ Dendrimers: therapeutic use / Humans / Nanomedicine /
Prion Diseases: drug therapy / Prion Diseases: metabolism /
Prions: antagonists $\&$ inhibitors / Prions: chemistry /
Prions: metabolism / Protein Conformation: drug effects /
Dendrimers (NLM Chemicals) / Prions (NLM Chemicals)},
cin = {Ext AG Tatzelt},
ddc = {570},
cid = {I:(DE-2719)5000053},
pnm = {342 - Disease Mechanisms and Model Systems (POF3-342)},
pid = {G:(DE-HGF)POF3-342},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:23764833},
pmc = {pmc:PMC3783103},
doi = {10.4161/pri.24431},
url = {https://pub.dzne.de/record/137108},
}