PolyQ-expanded ataxin-3 protein levels in peripheral blood mononuclear cells correlate with clinical parameters in SCA3: a pilot study

In view of upcoming clinical trials, quantitative molecular markers accessible in peripheral blood are of critical importance as prognostic or pharmacodynamic markers in genetic neurodegenerative diseases such as Spinocerebellar Ataxia Type 3 (SCA3), in particular for signaling target engagement. In...

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Pubblicato in:J Neurol
Autori principali: Gonsior, Kathrin, Kaucher, Gabriele Anna, Pelz, Patrik, Schumann, Dorothea, Gansel, Melanie, Kuhs, Sandra, Klockgether, Thomas, Forlani, Sylvie, Durr, Alexandra, Hauser, Stefan, Rattay, Tim W., Synofzik, Matthis, Hengel, Holger, Schöls, Ludger, Rieß, Olaf H., Hübener-Schmid, Jeannette
Natura: Artigo
Lingua:Inglês
Pubblicazione: Springer Berlin Heidelberg 2020
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Original Communication
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC7990753/
https://ncbi.nlm.nih.gov/pubmed/33106888
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s00415-020-10274-y
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