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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. Among authors: motlik j. Sci Transl Med. 2021 Apr 7;13(588):eabb8920. doi: 10.1126/scitranslmed.abb8920. Sci Transl Med. 2021. PMID: 33827977
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: motlik j. 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.
Transduction profiles in minipig following MRI guided delivery of AAV-5 into thalamic and corona radiata areas.
Pietersz KL, Pouw S, Klima J, Ellederova Z, Bohuslavova B, Chrastina J, Liscak R, Urgosik D, Starek Z, Crha M, Lewis O, Wooley M, Johnson D, Brouwers CC, Evers M, Motlik J, Martens GJM, Konstantinova PS, Blits B. Pietersz KL, et al. Among authors: motlik j. J Neurosci Methods. 2022 Jan 1;365:109382. doi: 10.1016/j.jneumeth.2021.109382. Epub 2021 Oct 9. J Neurosci Methods. 2022. PMID: 34637809
Mutated Huntingtin Causes Testicular Pathology in Transgenic Minipig Boars.
Macakova M, Bohuslavova B, Vochozkova P, Pavlok A, Sedlackova M, Vidinska D, Vochyanova K, Liskova I, Valekova I, Baxa M, Ellederova Z, Klima J, Juhas S, Juhasova J, Klouckova J, Haluzik M, Klempir J, Hansikova H, Spacilova J, Collins R, Blumenthal I, Talkowski M, Gusella JF, Howland DS, DiFiglia M, Motlik J. Macakova M, et al. Among authors: motlik j. Neurodegener Dis. 2016;16(3-4):245-59. doi: 10.1159/000443665. Epub 2016 Mar 10. Neurodegener Dis. 2016. PMID: 26959244
Mitochondrial Metabolism in a Large-Animal Model of Huntington Disease: The Hunt for Biomarkers in the Spermatozoa of Presymptomatic Minipigs.
Krizova J, Stufkova H, Rodinova M, Macakova M, Bohuslavova B, Vidinska D, Klima J, Ellederova Z, Pavlok A, Howland DS, Zeman J, Motlik J, Hansikova H. Krizova J, et al. Among authors: motlik j. Neurodegener Dis. 2017;17(4-5):213-226. doi: 10.1159/000475467. Epub 2017 Jun 21. Neurodegener Dis. 2017. PMID: 28633139
Development of an AAV-Based MicroRNA Gene Therapy to Treat Machado-Joseph Disease.
Martier R, Sogorb-Gonzalez M, Stricker-Shaver J, Hübener-Schmid J, Keskin S, Klima J, Toonen LJ, Juhas S, Juhasova J, Ellederova Z, Motlik J, Haas E, van Deventer S, Konstantinova P, Nguyen HP, Evers MM. Martier R, et al. Among authors: motlik j. Mol Ther Methods Clin Dev. 2019 Oct 28;15:343-358. doi: 10.1016/j.omtm.2019.10.008. eCollection 2019 Dec 13. Mol Ther Methods Clin Dev. 2019. PMID: 31828177 Free PMC article.
157 results