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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.
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.
Targeting RNA-Mediated Toxicity in C9orf72 ALS and/or FTD by RNAi-Based Gene Therapy.
Martier R, Liefhebber JM, García-Osta A, Miniarikova J, Cuadrado-Tejedor M, Espelosin M, Ursua S, Petry H, van Deventer SJ, Evers MM, Konstantinova P. Martier R, et al. Among authors: van deventer sj. Mol Ther Nucleic Acids. 2019 Jun 7;16:26-37. doi: 10.1016/j.omtn.2019.02.001. Epub 2019 Feb 11. Mol Ther Nucleic Acids. 2019. PMID: 30825670 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.
Cerebral Organoids: A Human Model for AAV Capsid Selection and Therapeutic Transgene Efficacy in the Brain.
Depla JA, Sogorb-Gonzalez M, Mulder LA, Heine VM, Konstantinova P, van Deventer SJ, Wolthers KC, Pajkrt D, Sridhar A, Evers MM. Depla JA, et al. Among authors: van deventer sj. Mol Ther Methods Clin Dev. 2020 Jun 1;18:167-175. doi: 10.1016/j.omtm.2020.05.028. eCollection 2020 Sep 11. Mol Ther Methods Clin Dev. 2020. PMID: 32637448 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. 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.
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. 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.
436 results