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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: konstantinova p. Sci Transl Med. 2021 Apr 7;13(588):eabb8920. doi: 10.1126/scitranslmed.abb8920. Sci Transl Med. 2021. PMID: 33827977
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: konstantinova ps. 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
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: konstantinova p. 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 gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington's disease.
Miniarikova J, Zimmer V, Martier R, Brouwers CC, Pythoud C, Richetin K, Rey M, Lubelski J, Evers MM, van Deventer SJ, Petry H, Déglon N, Konstantinova P. Miniarikova J, et al. Among authors: konstantinova p. Gene Ther. 2017 Oct;24(10):630-639. doi: 10.1038/gt.2017.71. Epub 2017 Aug 3. Gene Ther. 2017. PMID: 28771234 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: konstantinova p. 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.
Potent and sustained huntingtin lowering via AAV5 encoding miRNA preserves striatal volume and cognitive function in a humanized mouse model of Huntington disease.
Caron NS, Southwell AL, Brouwers CC, Cengio LD, Xie Y, Black HF, Anderson LM, Ko S, Zhu X, van Deventer SJ, Evers MM, Konstantinova P, Hayden MR. Caron NS, et al. Among authors: konstantinova p. Nucleic Acids Res. 2020 Jan 10;48(1):36-54. doi: 10.1093/nar/gkz976. Nucleic Acids Res. 2020. PMID: 31745548 Free PMC article.
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: konstantinova p. 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.
44 results