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Nobre RJ, Lobo DD, Henriques C, et al. MiRNA-Mediated Knockdown of ATXN3 Alleviates Molecular Disease Hallmarks in a Mouse Model for Spinocerebellar Ataxia Type 3. Nucleic Acid Ther. 2021;doi: 10.1089/nat.2021.0020.
Nobre, R. J., Lobo, D. D., Henriques, C., Duarte, S. P., Lopes, S. M., Silva, A. C., Lopes, M. M., Mariet, F., Schwarz, L. K., Baatje, M. S., Ferreira, V., Vallès, A., Pereira de Almeida, L., Evers, M. M., & Toonen, L. J. A. (2021). MiRNA-Mediated Knockdown of ATXN3 Alleviates Molecular Disease Hallmarks in a Mouse Model for Spinocerebellar Ataxia Type 3. Nucleic acid therapeutics, . https://doi.org/10.1089/nat.2021.0020
Nobre, Rui Jorge, et al. "MiRNA-Mediated Knockdown of ATXN3 Alleviates Molecular Disease Hallmarks in a Mouse Model for Spinocerebellar Ataxia Type 3." Nucleic acid therapeutics vol. (2021). doi: https://doi.org/10.1089/nat.2021.0020
Nobre RJ, Lobo DD, Henriques C, Duarte SP, Lopes SM, Silva AC, Lopes MM, Mariet F, Schwarz LK, Baatje MS, Ferreira V, Vallès A, Pereira de Almeida L, Evers MM, Toonen LJA. MiRNA-Mediated Knockdown of ATXN3 Alleviates Molecular Disease Hallmarks in a Mouse Model for Spinocerebellar Ataxia Type 3. Nucleic Acid Ther. 2021 Dec 07; doi: 10.1089/nat.2021.0020. Epub 2021 Dec 07. PMID: 34878314.
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Nucleic Acid Ther. 2021 Dec 07; doi: 10.1089/nat.2021.0020. Epub 2021 Dec 07.

MiRNA-Mediated Knockdown of ATXN3 Alleviates Molecular Disease Hallmarks in a Mouse Model for Spinocerebellar Ataxia Type 3.

Nucleic acid therapeutics

Rui Jorge Nobre, Diana D Lobo, Carina Henriques, Sonia P Duarte, Sara M Lopes, Ana C Silva, Miguel M Lopes, Fanny Mariet, Lukas K Schwarz, M S Baatje, Valerie Ferreira, Astrid Vallès, Luis Pereira de Almeida, Melvin M Evers, Lodewijk J A Toonen

Affiliations

  1. Center for Neuroscience and Cell Biology (CNC), Molecular Therapy of Brain Disorders Group, University of Coimbra, Coimbra, Portugal.
  2. Center for Innovative Biomedicine and Biotechnology (CIBB), Vectors, Gene and Cell Therapy Group, University of Coimbra, Coimbra, Portugal.
  3. ViraVector-Viral Vector for Gene Transfer Core Facility and University of Coimbra, Coimbra, Portugal.
  4. Institute for Interdisciplinary Research (III), University of Coimbra, Coimbra, Portugal.
  5. Faculty of Pharmacy, University of Coimbra, Coimbra, Portugal.
  6. uniQure Biopharma b.v., Amsterdam, the Netherlands.

PMID: 34878314 DOI: 10.1089/nat.2021.0020

Abstract

Spinocerebellar ataxia type 3 (SCA3) is a neurodegenerative disorder caused by the expansion of a CAG repeat in the

Keywords: AAV; ATXN3; ataxin-3; gene therapy; miRNA; spinocerebellar ataxia type 3

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