Home > Publications Database > Polyglutamine-Expanded Ataxin-3: A Target Engagement Marker for Spinocerebellar Ataxia Type 3 in Peripheral Blood. > print

001     162615
005     20240425115930.0
024 7 _ |a 10.1002/mds.28749
|2 doi
024 7 _ |a pmid:34397117
|2 pmid
024 7 _ |a 0885-3185
|2 ISSN
024 7 _ |a 1531-8257
|2 ISSN
024 7 _ |a altmetric:111895599
|2 altmetric
037 _ _ |a DZNE-2021-01311
041 _ _ |a English
082 _ _ |a 610
100 1 _ |a Hübener-Schmid, Jeannette
|0 0000-0002-4973-0923
|b 0
245 _ _ |a Polyglutamine-Expanded Ataxin-3: A Target Engagement Marker for Spinocerebellar Ataxia Type 3 in Peripheral Blood.
260 _ _ |a New York, NY
|c 2021
|b Wiley
336 7 _ |a article
|2 DRIVER
336 7 _ |a Output Types/Journal article
|2 DataCite
336 7 _ |a Journal Article
|b journal
|m journal
|0 PUB:(DE-HGF)16
|s 1713964740_26793
|2 PUB:(DE-HGF)
336 7 _ |a ARTICLE
|2 BibTeX
336 7 _ |a JOURNAL_ARTICLE
|2 ORCID
336 7 _ |a Journal Article
|0 0
|2 EndNote
520 _ _ |a Spinocerebellar 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.
536 _ _ |a 353 - Clinical and Health Care Research (POF4-353)
|0 G:(DE-HGF)POF4-353
|c POF4-353
|f POF IV
|x 0
588 _ _ |a Dataset connected to CrossRef, PubMed, , Journals: pub.dzne.de
650 _ 7 |a ataxin-3; Machado-Joseph disease; spinocerebellar ataxia type 3; singulex technology; target engagement biomarker
|2 Other
650 _ 2 |a Ataxin-3: genetics
|2 MeSH
650 _ 2 |a Cross-Sectional Studies
|2 MeSH
650 _ 2 |a Humans
|2 MeSH
650 _ 2 |a Machado-Joseph Disease: drug therapy
|2 MeSH
650 _ 2 |a Machado-Joseph Disease: genetics
|2 MeSH
650 _ 2 |a Neurodegenerative Diseases
|2 MeSH
650 _ 2 |a Peptides
|2 MeSH
693 _ _ |0 EXP:(DE-2719)ESMI-20140101
|5 EXP:(DE-2719)ESMI-20140101
|e European Spinocerebellar Ataxia Type 3/Machado-Joseph Disease Initiative
|x 0
700 1 _ |a Kuhlbrodt, Kirsten
|b 1
700 1 _ |a Peladan, Julien
|b 2
700 1 _ |a Faber, Jennifer
|0 P:(DE-2719)2811327
|b 3
|u dzne
700 1 _ |a Santana, Magda M
|b 4
700 1 _ |a Hengel, Holger
|0 P:(DE-2719)2811940
|b 5
|u dzne
700 1 _ |a Jacobi, Heike
|0 P:(DE-HGF)0
|b 6
700 1 _ |a Reetz, Kathrin
|0 0000-0002-9730-9228
|b 7
700 1 _ |a Garcia-Moreno, Hector
|b 8
700 1 _ |a Raposo, Mafalda
|0 0000-0001-6899-0352
|b 9
700 1 _ |a van Gaalen, Judith
|b 10
700 1 _ |a Infante, Jon
|0 0000-0003-4025-4606
|b 11
700 1 _ |a Steiner, Katharina M
|b 12
700 1 _ |a de Vries, Jeroen
|b 13
700 1 _ |a Verbeek, Marcel M
|b 14
700 1 _ |a Giunti, Paola
|b 15
700 1 _ |a Pereira de Almeida, Luis
|0 0000-0001-5831-3307
|b 16
700 1 _ |a Lima, Manuela
|b 17
700 1 _ |a van de Warrenburg, Bart
|b 18
700 1 _ |a Schöls, Ludger
|0 P:(DE-2719)2810795
|b 19
|u dzne
700 1 _ |a Klockgether, Thomas
|0 P:(DE-2719)2810314
|b 20
|u dzne
700 1 _ |a Synofzik, Matthis
|0 P:(DE-2719)2811275
|b 21
|u dzne
700 1 _ |a Initiative, European Spinocerebellar Ataxia Type-3/Machado-Joseph Disease
|b 22
|e Collaboration Author
700 1 _ |a Riess, Olaf
|b 23
773 _ _ |a 10.1002/mds.28749
|g Vol. 36, no. 11, p. 2675 - 2681
|0 PERI:(DE-600)2041249-6
|n 11
|p 2675 - 2681
|t Movement disorders
|v 36
|y 2021
|x 1531-8257
856 4 _ |y OpenAccess
|u https://pub.dzne.de/record/162615/files/DZNE-2021-01311.pdf
856 4 _ |y OpenAccess
|x pdfa
|u https://pub.dzne.de/record/162615/files/DZNE-2021-01311.pdf?subformat=pdfa
909 C O |o oai:pub.dzne.de:162615
|p openaire
|p open_access
|p VDB
|p driver
|p dnbdelivery
910 1 _ |a Deutsches Zentrum für Neurodegenerative Erkrankungen
|0 I:(DE-588)1065079516
|k DZNE
|b 3
|6 P:(DE-2719)2811327
910 1 _ |a Deutsches Zentrum für Neurodegenerative Erkrankungen
|0 I:(DE-588)1065079516
|k DZNE
|b 5
|6 P:(DE-2719)2811940
910 1 _ |a Deutsches Zentrum für Neurodegenerative Erkrankungen
|0 I:(DE-588)1065079516
|k DZNE
|b 19
|6 P:(DE-2719)2810795
910 1 _ |a Deutsches Zentrum für Neurodegenerative Erkrankungen
|0 I:(DE-588)1065079516
|k DZNE
|b 20
|6 P:(DE-2719)2810314
910 1 _ |a Deutsches Zentrum für Neurodegenerative Erkrankungen
|0 I:(DE-588)1065079516
|k DZNE
|b 21
|6 P:(DE-2719)2811275
913 1 _ |a DE-HGF
|b Gesundheit
|l Neurodegenerative Diseases
|1 G:(DE-HGF)POF4-350
|0 G:(DE-HGF)POF4-353
|3 G:(DE-HGF)POF4
|2 G:(DE-HGF)POF4-300
|4 G:(DE-HGF)POF
|v Clinical and Health Care Research
|x 0
914 1 _ |y 2021
915 _ _ |a DBCoverage
|0 StatID:(DE-HGF)0200
|2 StatID
|b SCOPUS
|d 2022-11-11
915 _ _ |a DBCoverage
|0 StatID:(DE-HGF)0300
|2 StatID
|b Medline
|d 2022-11-11
915 _ _ |a DBCoverage
|0 StatID:(DE-HGF)1030
|2 StatID
|b Current Contents - Life Sciences
|d 2022-11-11
915 _ _ |a DBCoverage
|0 StatID:(DE-HGF)0600
|2 StatID
|b Ebsco Academic Search
|d 2022-11-11
915 _ _ |a JCR
|0 StatID:(DE-HGF)0100
|2 StatID
|b MOVEMENT DISORD : 2021
|d 2022-11-11
915 _ _ |a Creative Commons Attribution-NonCommercial CC BY-NC 4.0
|0 LIC:(DE-HGF)CCBYNC4
|2 HGFVOC
915 _ _ |a DEAL Wiley
|0 StatID:(DE-HGF)3001
|2 StatID
|d 2021-01-30
|w ger
915 _ _ |a WoS
|0 StatID:(DE-HGF)0113
|2 StatID
|b Science Citation Index Expanded
|d 2021-01-30
915 _ _ |a DBCoverage
|0 StatID:(DE-HGF)0150
|2 StatID
|b Web of Science Core Collection
|d 2022-11-11
915 _ _ |a OpenAccess
|0 StatID:(DE-HGF)0510
|2 StatID
915 _ _ |a Peer Review
|0 StatID:(DE-HGF)0030
|2 StatID
|b ASC
|d 2022-11-11
915 _ _ |a IF >= 5
|0 StatID:(DE-HGF)9905
|2 StatID
|b MOVEMENT DISORD : 2021
|d 2022-11-11
915 _ _ |a DBCoverage
|0 StatID:(DE-HGF)0160
|2 StatID
|b Essential Science Indicators
|d 2021-01-30
915 _ _ |a DBCoverage
|0 StatID:(DE-HGF)1110
|2 StatID
|b Current Contents - Clinical Medicine
|d 2022-11-11
915 _ _ |a Nationallizenz
|0 StatID:(DE-HGF)0420
|2 StatID
|d 2022-11-11
|w ger
915 _ _ |a DBCoverage
|0 StatID:(DE-HGF)0199
|2 StatID
|b Clarivate Analytics Master Journal List
|d 2022-11-11
920 1 _ |0 I:(DE-2719)1210000
|k AG Gasser
|l Parkinson Genetics
|x 0
920 1 _ |0 I:(DE-2719)1011101
|k Patient Studies Bonn
|l Patient Studies Bonn
|x 1
920 1 _ |0 I:(DE-2719)1011001
|k AG Klockgether
|l Patient Studies
|x 2
980 _ _ |a journal
980 _ _ |a VDB
980 _ _ |a UNRESTRICTED
980 _ _ |a I:(DE-2719)1210000
980 _ _ |a I:(DE-2719)1011101
980 _ _ |a I:(DE-2719)1011001
980 1 _ |a FullTexts

LibraryCollectionCLSMajorCLSMinorLanguageAuthor
Marc 21