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.jpg): Failed to open stream: No such file or directory in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 117 Transl+Neurodegener
2017 ; 6
(ä): 6
Nephropedia Template TP
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English Wikipedia
Protein misfolding in neurodegenerative diseases: implications and strategies
#MMPMID28293421
Sweeney P
; Park H
; Baumann M
; Dunlop J
; Frydman J
; Kopito R
; McCampbell A
; Leblanc G
; Venkateswaran A
; Nurmi A
; Hodgson R
Transl Neurodegener
2017[]; 6
(ä): 6
PMID28293421
show ga
A hallmark of neurodegenerative proteinopathies is the formation of misfolded
protein aggregates that cause cellular toxicity and contribute to cellular
proteostatic collapse. Therapeutic options are currently being explored that
target different steps in the production and processing of proteins implicated in
neurodegenerative disease, including synthesis, chaperone-assisted folding and
trafficking, and degradation via the proteasome and autophagy pathways. Other
therapies, like mTOR inhibitors and activators of the heat shock response, can
rebalance the entire proteostatic network. However, there are major challenges
that impact the development of novel therapies, including incomplete knowledge of
druggable disease targets and their mechanism of action as well as a lack of
biomarkers to monitor disease progression and therapeutic response. A notable
development is the creation of collaborative ecosystems that include patients,
clinicians, basic and translational researchers, foundations and regulatory
agencies to promote scientific rigor and clinical data to accelerate the
development of therapies that prevent, reverse or delay the progression of
neurodegenerative proteinopathies.