Spinocerebellar ataxia type 3: from pathogenesis to promising therapeutics

Ana Carolina Silva; Miguel Monteiro Lopes; Diana Duarte Lobo; Carina Henriques; Luís Pereira de Almeida; Rui Jorge Nobre

doi:10.1016/j.molmed.2025.06.007

Spinocerebellar ataxia type 3: from pathogenesis to promising therapeutics

Trends Mol Med. 2025 Nov;31(11):1065-1066. doi: 10.1016/j.molmed.2025.06.007. Epub 2025 Jul 23.

Authors

Ana Carolina Silva¹, Miguel Monteiro Lopes¹, Diana Duarte Lobo¹, Carina Henriques², Luís Pereira de Almeida³, Rui Jorge Nobre⁴

Affiliations

¹ CNC-UC - Center for Neuroscience and Cell Biology, University of Coimbra, Coimbra, Portugal; CIBB - Center for Innovative Biomedicine and Biotechnology, University of Coimbra, Coimbra, Portugal; Institute for Interdisciplinary Research, Doctoral Programme in Experimental Biology and Biomedicine (PDBEB), University of Coimbra, Coimbra, Portugal; GeneT - Gene Therapy Center of Excellence, University of Coimbra, Coimbra, Portugal.
² CNC-UC - Center for Neuroscience and Cell Biology, University of Coimbra, Coimbra, Portugal; CIBB - Center for Innovative Biomedicine and Biotechnology, University of Coimbra, Coimbra, Portugal; GeneT - Gene Therapy Center of Excellence, University of Coimbra, Coimbra, Portugal; FFUC - Faculty of Pharmacy, University of Coimbra, Coimbra, Portugal.
³ CNC-UC - Center for Neuroscience and Cell Biology, University of Coimbra, Coimbra, Portugal; CIBB - Center for Innovative Biomedicine and Biotechnology, University of Coimbra, Coimbra, Portugal; GeneT - Gene Therapy Center of Excellence, University of Coimbra, Coimbra, Portugal; FFUC - Faculty of Pharmacy, University of Coimbra, Coimbra, Portugal; ViraVector - Viral Vector for Gene Transfer Core Facility, University of Coimbra, Coimbra, Portugal. Electronic address: luispa@cnc.uc.pt.
⁴ CNC-UC - Center for Neuroscience and Cell Biology, University of Coimbra, Coimbra, Portugal; CIBB - Center for Innovative Biomedicine and Biotechnology, University of Coimbra, Coimbra, Portugal; GeneT - Gene Therapy Center of Excellence, University of Coimbra, Coimbra, Portugal; ViraVector - Viral Vector for Gene Transfer Core Facility, University of Coimbra, Coimbra, Portugal; III-UC - Institute for Interdisciplinary Research, University of Coimbra, Coimbra, Portugal. Electronic address: rui.nobre@cnc.uc.pt.

PMID: 40707300
DOI: 10.1016/j.molmed.2025.06.007

No abstract available

Keywords: Machado-Joseph disease; gene silencing; polyglutamine disorders; spinocerebellar ataxia type 3; therapeutics.