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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: sogorb gonzalez m. Sci Transl Med. 2021 Apr 7;13(588):eabb8920. doi: 10.1126/scitranslmed.abb8920. Sci Transl Med. 2021. PMID: 33827977
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: sogorb gonzalez m. 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.
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: sogorb gonzalez m. 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.
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: sogorb gonzalez m. 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.