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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Vydáno v:J Neurol
Hlavní autoři: 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
Médium: Artigo
Jazyk:Inglês
Vydáno: Springer Berlin Heidelberg 2020
Témata:
Original Communication
On-line přístup: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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