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AAV-Mediated CAG-Targeting Selectively Reduces Polyglutamine-Expanded Protein and Attenuates Disease Phenotypes in a Spinocerebellar Ataxia Mouse Model.
Niewiadomska-Cimicka A, Fievet L, Surdyka M, Jesion E, Keime C, Singer E, Eisenmann A, Kalinowska-Poska Z, Nguyen HHP, Fiszer A, Figiel M, Trottier Y. Niewiadomska-Cimicka A, et al. Among authors: fiszer a. Int J Mol Sci. 2024 Apr 15;25(8):4354. doi: 10.3390/ijms25084354. Int J Mol Sci. 2024. PMID: 38673939 Free PMC article.
Insights into RNA-mediated pathology in new mouse models of Huntington's disease.
Wozna-Wysocka M, Jazurek-Ciesiolka M, Przybyl L, Wronka D, Misiorek JO, Suszynska-Zajczyk J, Figura G, Ciesiolka A, Sobieszczanska P, Zeller A, Niemira M, Switonski PM, Fiszer A. Wozna-Wysocka M, et al. Among authors: fiszer a. FASEB J. 2024 Dec 15;38(23):e70182. doi: 10.1096/fj.202401465R. FASEB J. 2024. PMID: 39604147 Free PMC article.
CAG-targeted brain-permeable therapy tested in biallelic humanized polyQ mouse models.
Surdyka M, Kalinowska-Pośka Ż, Niewiadomska-Cimicka A, Jesion E, Fiszer A, Singer-Mikosch E, Fievet L, Przybyl L, Caron NS, Hayden MR, Nguyen HP, Trottier Y, Figiel M. Surdyka M, et al. Among authors: fiszer a. Mol Ther Nucleic Acids. 2025 Feb 22;36(2):102496. doi: 10.1016/j.omtn.2025.102496. eCollection 2025 Jun 10. Mol Ther Nucleic Acids. 2025. PMID: 40171277 Free PMC article.
38 results