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000162615 1001_ $$00000-0002-4973-0923$$aHübener-Schmid, Jeannette$$b0
000162615 245__ $$aPolyglutamine-Expanded Ataxin-3: A Target Engagement Marker for Spinocerebellar Ataxia Type 3 in Peripheral Blood.
000162615 260__ $$aNew York, NY$$bWiley$$c2021
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000162615 520__ $$aSpinocerebellar ataxia type 3 is a rare neurodegenerative disease caused by a CAG repeat expansion in the ataxin-3 gene. Although no curative therapy is yet available, preclinical gene-silencing approaches to reduce polyglutamine (polyQ) toxicity demonstrate promising results. In view of upcoming clinical trials, quantitative and easily accessible molecular markers are of critical importance as pharmacodynamic and particularly as target engagement markers.We aimed at developing an ultrasensitive immunoassay to measure specifically polyQ-expanded ataxin-3 in plasma and cerebrospinal fluid (CSF).Using the novel single molecule counting ataxin-3 immunoassay, we analyzed cross-sectional and longitudinal patient biomaterials.Statistical analyses revealed a correlation with clinical parameters and a stability of polyQ-expanded ataxin-3 during conversion from the pre-ataxic to the ataxic phases.The novel immunoassay is able to quantify polyQ-expanded ataxin-3 in plasma and CSF, whereas ataxin-3 levels in plasma correlate with disease severity. Longitudinal analyses demonstrated a high stability of polyQ-expanded ataxin-3 over a short period. © 2021 The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.
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000162615 650_7 $$2Other$$aataxin-3; Machado-Joseph disease; spinocerebellar ataxia type 3; singulex technology; target engagement biomarker
000162615 650_2 $$2MeSH$$aAtaxin-3: genetics
000162615 650_2 $$2MeSH$$aCross-Sectional Studies
000162615 650_2 $$2MeSH$$aHumans
000162615 650_2 $$2MeSH$$aMachado-Joseph Disease: drug therapy
000162615 650_2 $$2MeSH$$aMachado-Joseph Disease: genetics
000162615 650_2 $$2MeSH$$aNeurodegenerative Diseases
000162615 650_2 $$2MeSH$$aPeptides
000162615 693__ $$0EXP:(DE-2719)ESMI-20140101$$5EXP:(DE-2719)ESMI-20140101$$eEuropean Spinocerebellar Ataxia Type 3/Machado-Joseph Disease Initiative$$x0
000162615 7001_ $$aKuhlbrodt, Kirsten$$b1
000162615 7001_ $$aPeladan, Julien$$b2
000162615 7001_ $$0P:(DE-2719)2811327$$aFaber, Jennifer$$b3$$udzne
000162615 7001_ $$aSantana, Magda M$$b4
000162615 7001_ $$0P:(DE-2719)2811940$$aHengel, Holger$$b5$$udzne
000162615 7001_ $$0P:(DE-HGF)0$$aJacobi, Heike$$b6
000162615 7001_ $$00000-0002-9730-9228$$aReetz, Kathrin$$b7
000162615 7001_ $$aGarcia-Moreno, Hector$$b8
000162615 7001_ $$00000-0001-6899-0352$$aRaposo, Mafalda$$b9
000162615 7001_ $$avan Gaalen, Judith$$b10
000162615 7001_ $$00000-0003-4025-4606$$aInfante, Jon$$b11
000162615 7001_ $$aSteiner, Katharina M$$b12
000162615 7001_ $$ade Vries, Jeroen$$b13
000162615 7001_ $$aVerbeek, Marcel M$$b14
000162615 7001_ $$aGiunti, Paola$$b15
000162615 7001_ $$00000-0001-5831-3307$$aPereira de Almeida, Luis$$b16
000162615 7001_ $$aLima, Manuela$$b17
000162615 7001_ $$avan de Warrenburg, Bart$$b18
000162615 7001_ $$0P:(DE-2719)2810795$$aSchöls, Ludger$$b19$$udzne
000162615 7001_ $$0P:(DE-2719)2810314$$aKlockgether, Thomas$$b20$$udzne
000162615 7001_ $$0P:(DE-2719)2811275$$aSynofzik, Matthis$$b21$$udzne
000162615 7001_ $$aInitiative, European Spinocerebellar Ataxia Type-3/Machado-Joseph Disease$$b22$$eCollaboration Author
000162615 7001_ $$aRiess, Olaf$$b23
000162615 773__ $$0PERI:(DE-600)2041249-6$$a10.1002/mds.28749$$gVol. 36, no. 11, p. 2675 - 2681$$n11$$p2675 - 2681$$tMovement disorders$$v36$$x1531-8257$$y2021
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