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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: blits b. 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: blits b. 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: blits b. 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
Embedding siRNA sequences targeting apolipoprotein B100 in shRNA and miRNA scaffolds results in differential processing and in vivo efficacy.
Maczuga P, Lubelski J, van Logtenstein R, Borel F, Blits B, Fakkert E, Costessi A, Butler D, van Deventer S, Petry H, Koornneef A, Konstantinova P. Maczuga P, et al. Among authors: blits b. Mol Ther. 2013 Jan;21(1):217-27. doi: 10.1038/mt.2012.160. Epub 2012 Oct 23. Mol Ther. 2013. PMID: 23089734 Free PMC article.
Therapeutic expression of hairpins targeting apolipoprotein B100 induces phenotypic and transcriptome changes in murine liver.
Maczuga P, Verheij J, van der Loos C, van Logtenstein R, Hooijer G, Martier R, Borel F, Lubelski J, Koornneef A, Blits B, van Deventer S, Petry H, Konstantinova P. Maczuga P, et al. Among authors: blits b. Gene Ther. 2014 Jan;21(1):60-70. doi: 10.1038/gt.2013.58. Epub 2013 Oct 24. Gene Ther. 2014. PMID: 24152580 Free PMC article.
48 results