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miRNA-Mediated Knockdown of ATXN3 Alleviates Molecular Disease Hallmarks in a Mouse Model for Spinocerebellar Ataxia Type 3.
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. Nobre RJ, et al. Among authors: valles a. Nucleic Acid Ther. 2022 Jun;32(3):194-205. doi: 10.1089/nat.2021.0020. Epub 2021 Dec 7. Nucleic Acid Ther. 2022. PMID: 34878314 Free PMC article.
AAV5-miHTT Gene Therapy Demonstrates Broad Distribution and Strong Human Mutant Huntingtin Lowering in a Huntington's Disease Minipig Model.
Evers MM, Miniarikova J, Juhas S, Vallès A, Bohuslavova B, Juhasova J, Skalnikova HK, Vodicka P, Valekova I, Brouwers C, Blits B, Lubelski J, Kovarova H, Ellederova Z, van Deventer SJ, Petry H, Motlik J, Konstantinova P. Evers MM, et al. Among authors: valles a. Mol Ther. 2018 Sep 5;26(9):2163-2177. doi: 10.1016/j.ymthe.2018.06.021. Epub 2018 Jun 25. Mol Ther. 2018. PMID: 30007561 Free PMC article.
AAV5-miHTT Lowers Huntingtin mRNA and Protein without Off-Target Effects in Patient-Derived Neuronal Cultures and Astrocytes.
Keskin S, Brouwers CC, Sogorb-Gonzalez M, Martier R, Depla JA, Vallès A, van Deventer SJ, Konstantinova P, Evers MM. Keskin S, et al. Among authors: valles a. Mol Ther Methods Clin Dev. 2019 Oct 4;15:275-284. doi: 10.1016/j.omtm.2019.09.010. eCollection 2019 Dec 13. Mol Ther Methods Clin Dev. 2019. PMID: 31737741 Free PMC article.
Intrastriatal Administration of AAV5-miHTT in Non-Human Primates and Rats Is Well Tolerated and Results in miHTT Transgene Expression in Key Areas of Huntington Disease Pathology.
Spronck EA, Vallès A, Lampen MH, Montenegro-Miranda PS, Keskin S, Heijink L, Evers MM, Petry H, Deventer SJV, Konstantinova P, Haan M. Spronck EA, et al. Among authors: valles a. Brain Sci. 2021 Jan 20;11(2):129. doi: 10.3390/brainsci11020129. Brain Sci. 2021. PMID: 33498212 Free PMC article.
Widespread and sustained target engagement in Huntington's disease minipigs upon intrastriatal microRNA-based gene therapy.
Vallès A, Evers MM, Stam A, Sogorb-Gonzalez M, Brouwers C, Vendrell-Tornero C, Acar-Broekmans S, Paerels L, Klima J, Bohuslavova B, Pintauro R, Fodale V, Bresciani A, Liscak R, Urgosik D, Starek Z, Crha M, Blits B, Petry H, Ellederova Z, Motlik J, van Deventer S, Konstantinova P. Vallès A, et al. Sci Transl Med. 2021 Apr 7;13(588):eabb8920. doi: 10.1126/scitranslmed.abb8920. Sci Transl Med. 2021. PMID: 33827977
Secreted therapeutics: monitoring durability of microRNA-based gene therapies in the central nervous system.
Sogorb-Gonzalez M, Vendrell-Tornero C, Snapper J, Stam A, Keskin S, Miniarikova J, Spronck EA, de Haan M, Nieuwland R, Konstantinova P, van Deventer SJ, Evers MM, Vallès A. Sogorb-Gonzalez M, et al. Among authors: valles a. Brain Commun. 2021 Apr 1;3(2):fcab054. doi: 10.1093/braincomms/fcab054. eCollection 2021. Brain Commun. 2021. PMID: 34704020 Free PMC article.
Proof-of-concept study for liver-directed miQURE technology in a dyslipidemic mouse model.
Zancanella V, Vallès A, Liefhebber JMP, Paerels L, Tornero CV, Wattimury H, van der Zon T, van Rooijen K, Golinska M, Grevelink T, Ehlert E, Pieterman EJ, Keijzer N, Princen HMG, Stokman G, Liu YP. Zancanella V, et al. Among authors: valles a. Mol Ther Nucleic Acids. 2023 Apr 7;32:454-467. doi: 10.1016/j.omtn.2023.04.004. eCollection 2023 Jun 13. Mol Ther Nucleic Acids. 2023. PMID: 37168797 Free PMC article.
161 results