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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.
Transduction patterns in the CNS following various routes of AAV-5-mediated gene delivery.
Pietersz KL, Martier RM, Baatje MS, Liefhebber JM, Brouwers CC, Pouw SM, Fokkert L, Lubelski J, Petry H, Martens GJM, van Deventer SJ, Konstantinova P, Blits B. Pietersz KL, et al. Among authors: van deventer sj. Gene Ther. 2021 Aug;28(7-8):435-446. doi: 10.1038/s41434-020-0178-0. Epub 2020 Aug 15. Gene Ther. 2021. PMID: 32801344
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 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.
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
Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington's Disease.
Miniarikova J, Zanella I, Huseinovic A, van der Zon T, Hanemaaijer E, Martier R, Koornneef A, Southwell AL, Hayden MR, van Deventer SJ, Petry H, Konstantinova P. Miniarikova J, et al. Among authors: van deventer sj, van der zon t. Mol Ther Nucleic Acids. 2016 Mar 22;5(3):e297. doi: 10.1038/mtna.2016.7. Mol Ther Nucleic Acids. 2016. PMID: 27003755 Free PMC article.
Artificial MicroRNAs Targeting C9orf72 Can Reduce Accumulation of Intra-nuclear Transcripts in ALS and FTD Patients.
Martier R, Liefhebber JM, Miniarikova J, van der Zon T, Snapper J, Kolder I, Petry H, van Deventer SJ, Evers MM, Konstantinova P. Martier R, et al. Among authors: van deventer sj, van der zon t. Mol Ther Nucleic Acids. 2019 Mar 1;14:593-608. doi: 10.1016/j.omtn.2019.01.010. Epub 2019 Jan 30. Mol Ther Nucleic Acids. 2019. PMID: 30776581 Free PMC article.
436 results