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@ARTICLE{HbenerSchmid:162615,
      author       = {Hübener-Schmid, Jeannette and Kuhlbrodt, Kirsten and
                      Peladan, Julien and Faber, Jennifer and Santana, Magda M and
                      Hengel, Holger and Jacobi, Heike and Reetz, Kathrin and
                      Garcia-Moreno, Hector and Raposo, Mafalda and van Gaalen,
                      Judith and Infante, Jon and Steiner, Katharina M and de
                      Vries, Jeroen and Verbeek, Marcel M and Giunti, Paola and
                      Pereira de Almeida, Luis and Lima, Manuela and van de
                      Warrenburg, Bart and Schöls, Ludger and Klockgether, Thomas
                      and Synofzik, Matthis and Riess, Olaf},
      collaboration = {Initiative, European Spinocerebellar Ataxia
                      Type-3/Machado-Joseph Disease},
      title        = {{P}olyglutamine-{E}xpanded {A}taxin-3: {A} {T}arget
                      {E}ngagement {M}arker for {S}pinocerebellar {A}taxia {T}ype
                      3 in {P}eripheral {B}lood.},
      journal      = {Movement disorders},
      volume       = {36},
      number       = {11},
      issn         = {1531-8257},
      address      = {New York, NY},
      publisher    = {Wiley},
      reportid     = {DZNE-2021-01311},
      pages        = {2675 - 2681},
      year         = {2021},
      abstract     = {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.},
      keywords     = {Ataxin-3: genetics / Cross-Sectional Studies / Humans /
                      Machado-Joseph Disease: drug therapy / Machado-Joseph
                      Disease: genetics / Neurodegenerative Diseases / Peptides /
                      ataxin-3; Machado-Joseph disease; spinocerebellar ataxia
                      type 3; singulex technology; target engagement biomarker
                      (Other)},
      cin          = {AG Gasser / Patient Studies Bonn / AG Klockgether},
      ddc          = {610},
      cid          = {I:(DE-2719)1210000 / I:(DE-2719)1011101 /
                      I:(DE-2719)1011001},
      pnm          = {353 - Clinical and Health Care Research (POF4-353)},
      pid          = {G:(DE-HGF)POF4-353},
      experiment   = {EXP:(DE-2719)ESMI-20140101},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:34397117},
      doi          = {10.1002/mds.28749},
      url          = {https://pub.dzne.de/record/162615},
}