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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Bibliografske podrobnosti
izdano v:J Neurol
Main Authors: 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
Format: Artigo
Jezik:Inglês
Izdano: Springer Berlin Heidelberg 2020
Teme:
Original Communication
Online dostop: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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