MiRNA-Mediated Knockdown of ATXN3 Alleviates Molecular Disease Hallmarks in a Mouse Model for Spinocerebellar Ataxia Type 3-Reference-Cited by-同舟云学术

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

Published:2021-12-07 Issue: Volume: Page:
ISSN:2159-3337
Container-title:Nucleic Acid Therapeutics
language:en
Short-container-title:Nucleic Acid Therapeutics

Author:

Nobre Rui Jorge¹²³⁴^ORCID,Lobo Diana D.¹²⁴^ORCID,Henriques Carina¹²³⁵^ORCID,Duarte Sonia P.¹²⁴^ORCID,Lopes Sara M.¹²⁴^ORCID,Silva Ana C.¹²⁴^ORCID,Lopes Miguel M.¹²⁴^ORCID,Mariet Fanny⁶,Schwarz Lukas K.⁶,Baatje M.S.⁶,Ferreira Valerie⁶,Vallès Astrid⁶,Pereira de Almeida Luis¹²³⁵^ORCID,Evers Melvin M.⁶,Toonen Lodewijk J.A.⁶^ORCID

Affiliation:

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.

Publisher

Mary Ann Liebert Inc

Subject

Drug Discovery,Genetics,Molecular Biology,Molecular Medicine,Biochemistry

Link

https://www.liebertpub.com/doi/pdf/10.1089/nat.2021.0020

Reference72 articles.

1. Genetics, Mechanisms, and Therapeutic Progress in Polyglutamine Spinocerebellar Ataxias

2. Degenerative Ataxias: challenges in clinical research

3. Prediction of the age at onset in spinocerebellar ataxia type 1, 2, 3 and 6

4. Machado-joseph disease: Clinical, molecular, and metabolic characterization in chinese kindreds

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