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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: van deventer s. 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: van deventer sj. 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: van deventer sj. 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: van deventer sj. 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: van deventer sj. Nucleic Acids Res. 2020 Jan 10;48(1):36-54. doi: 10.1093/nar/gkz976. Nucleic Acids Res. 2020. PMID: 31745548 Free PMC article.
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: van deventer sj. Brain Commun. 2021 Apr 1;3(2):fcab054. doi: 10.1093/braincomms/fcab054. eCollection 2021. Brain Commun. 2021. PMID: 34704020 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: van deventer s. 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.
Mir-142-3p target sequences reduce transgene-directed immunogenicity following intramuscular adeno-associated virus 1 vector-mediated gene delivery.
Majowicz A, Maczuga P, Kwikkers KL, van der Marel S, van Logtenstein R, Petry H, van Deventer SJ, Konstantinova P, Ferreira V. Majowicz A, et al. Among authors: van der marel s, van deventer sj, van logtenstein r. J Gene Med. 2013 Jun-Jul;15(6-7):219-32. doi: 10.1002/jgm.2712. J Gene Med. 2013. PMID: 23658149
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: van deventer s, van logtenstein r. 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.
446 results