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Toward allele-specific targeting therapy and pharmacodynamic marker for spinocerebellar ataxia type 3.
Prudencio, Mercedes; Garcia-Moreno, Hector; Jansen-West, Karen R; Al-Shaikh, Rana Hanna; Gendron, Tania F; Heckman, Michael G; Spiegel, Matthew R; Carlomagno, Yari; Daughrity, Lillian M; Song, Yuping; Dunmore, Judith A; Byron, Natalie; Oskarsson, Björn; Nicholson, Katharine A; Staff, Nathan P; Gorcenco, Sorina; Puschmann, Andreas; Lemos, João; Januário, Cristina; LeDoux, Mark S; Friedman, Joseph H; Polke, James; Labrum, Robin; Shakkottai, Vikram; McLoughlin, Hayley S; Paulson, Henry L; Konno, Takuya; Onodera, Osamu; Ikeuchi, Takeshi; Tada, Mari; Kakita, Akiyoshi; Fryer, John D; Karremo, Christin; Gomes, Inês; Caviness, John N; Pittelkow, Mark R; Aasly, Jan; Pfeiffer, Ronald F; Veerappan, Venka; Eggenberger, Eric R; Freeman, William D; Huang, Josephine F; Uitti, Ryan J; Wierenga, Klaas J; Marin Collazo, Iris V; Tipton, Philip W; van Gerpen, Jay A; van Blitterswijk, Marka; Bu, Guojun; Wszolek, Zbigniew K.
Afiliação
  • Prudencio M; Department of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.
  • Garcia-Moreno H; Neuroscience Graduate Program, Mayo Clinic Graduate School of Biomedical Sciences, Jacksonville, FL 32224, USA.
  • Jansen-West KR; Ataxia Centre, Department of Clinical and Movement Neurosciences, UCL Queen Square Institute of Neurology, London WC1N 3BG, UK.
  • Al-Shaikh RH; Ataxia Centre, National Hospital for Neurology and Neurosurgery, University College London Hospitals NHS Trust, London WC1N 3BG, UK.
  • Gendron TF; Department of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.
  • Heckman MG; Department of Neurology, Mayo Clinic, Jacksonville, FL 32224, USA.
  • Spiegel MR; Department of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.
  • Carlomagno Y; Neuroscience Graduate Program, Mayo Clinic Graduate School of Biomedical Sciences, Jacksonville, FL 32224, USA.
  • Daughrity LM; Division of Biomedical Statistics and Informatics, Mayo Clinic, Jacksonville, FL 32224, USA.
  • Song Y; Division of Biomedical Statistics and Informatics, Mayo Clinic, Jacksonville, FL 32224, USA.
  • Dunmore JA; Department of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.
  • Byron N; Neuroscience Graduate Program, Mayo Clinic Graduate School of Biomedical Sciences, Jacksonville, FL 32224, USA.
  • Oskarsson B; Department of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.
  • Nicholson KA; Department of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.
  • Staff NP; Department of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.
  • Gorcenco S; Department of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.
  • Puschmann A; Department of Neurology, Mayo Clinic, Jacksonville, FL 32224, USA.
  • Lemos J; Sean M. Healey and AMG Center for ALS, Massachusetts General Hospital (MGH), Boston, MA 02114, USA.
  • Januário C; Department of Neurology, Mayo Clinic, Rochester, MN 55905, USA.
  • LeDoux MS; Lund University, Skåne University Hospital, Department of Clinical Sciences Lund, Neurology, Lund 22185, Sweden.
  • Friedman JH; Lund University, Skåne University Hospital, Department of Clinical Sciences Lund, Neurology, Lund 22185, Sweden.
  • Polke J; Coimbra University Hospital Centre, Coimbra University, Coimbra 3000-075, Portugal.
  • Labrum R; Coimbra University Hospital Centre, Coimbra University, Coimbra 3000-075, Portugal.
  • Shakkottai V; University of Memphis and Veracity Neuroscience LLC, Memphis, TN 38152, USA.
  • McLoughlin HS; Department of Neurology, Warren Alpert Medical School of Brown University, Providence, RI 02906, USA.
  • Paulson HL; Ataxia Centre, Department of Clinical and Movement Neurosciences, UCL Queen Square Institute of Neurology, London WC1N 3BG, UK.
  • Konno T; Ataxia Centre, National Hospital for Neurology and Neurosurgery, University College London Hospitals NHS Trust, London WC1N 3BG, UK.
  • Onodera O; Ataxia Centre, Department of Clinical and Movement Neurosciences, UCL Queen Square Institute of Neurology, London WC1N 3BG, UK.
  • Ikeuchi T; Ataxia Centre, National Hospital for Neurology and Neurosurgery, University College London Hospitals NHS Trust, London WC1N 3BG, UK.
  • Tada M; Department of Neurology, University of Michigan, Ann Arbor, MI 48109, USA.
  • Kakita A; Department of Neurology, University of Michigan, Ann Arbor, MI 48109, USA.
  • Fryer JD; Department of Neurology, University of Michigan, Ann Arbor, MI 48109, USA.
  • Karremo C; Department of Neurology, Brain Research Institute, Niigata University, Niigata 951-8585, Japan.
  • Gomes I; Department of Neurology, Brain Research Institute, Niigata University, Niigata 951-8585, Japan.
  • Caviness JN; Department of Molecular Genetics, Brain Research Institute, Niigata University, Niigata 951-8585, Japan.
  • Pittelkow MR; Department of Pathology, Brain Research Institute, Niigata University, Niigata 951-8585, Japan.
  • Aasly J; Department of Pathology, Brain Research Institute, Niigata University, Niigata 951-8585, Japan.
  • Pfeiffer RF; Neuroscience Graduate Program, Mayo Clinic Graduate School of Biomedical Sciences, Jacksonville, FL 32224, USA.
  • Veerappan V; Department of Neuroscience, Mayo Clinic, Scottsdale, AZ 85259, USA.
  • Eggenberger ER; Lund University, Skåne University Hospital, Department of Clinical Sciences Lund, Neurology, Lund 22185, Sweden.
  • Freeman WD; Coimbra University Hospital Centre, Coimbra University, Coimbra 3000-075, Portugal.
  • Huang JF; Department of Neurology, Mayo Clinic, Scottsdale, AZ 85259, USA.
  • Uitti RJ; Department of Dermatology, Mayo Clinic, Scottsdale, AZ 85259, USA.
  • Wierenga KJ; Norwegian University of Science and Technology, 7006 Trondheim, Norway.
  • Marin Collazo IV; Department of Neurology, Oregon Health & Science University, Portland, OR 97239, USA.
  • Tipton PW; Department of Neurology, Oregon Health & Science University, Portland, OR 97239, USA.
  • van Gerpen JA; Department of Neurology, Mayo Clinic, Jacksonville, FL 32224, USA.
  • van Blitterswijk M; Department of Neurology, Mayo Clinic, Jacksonville, FL 32224, USA.
  • Bu G; Department of Neurology, Mayo Clinic, Jacksonville, FL 32224, USA.
  • Wszolek ZK; Department of Neurology, Mayo Clinic, Jacksonville, FL 32224, USA.
Sci Transl Med ; 12(566)2020 10 21.
Article em En | MEDLINE | ID: mdl-33087504
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Doença de Machado-Joseph Tipo de estudo: Clinical_trials / Prognostic_studies Limite: Humans Idioma: En Ano de publicação: 2020 Tipo de documento: Article
Texto completo
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Doença de Machado-Joseph Tipo de estudo: Clinical_trials / Prognostic_studies Limite: Humans Idioma: En Ano de publicação: 2020 Tipo de documento: Article