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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: evers mm. 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.
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: evers mm. 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. 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.
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: evers mm. Nucleic Acids Res. 2020 Jan 10;48(1):36-54. doi: 10.1093/nar/gkz976. Nucleic Acids Res. 2020. PMID: 31745548 Free PMC article.
Intrastriatal Administration of AAV5-miHTT in Non-Human Primates and Rats Is Well Tolerated and Results in miHTT Transgene Expression in Key Areas of Huntington Disease Pathology.
Spronck EA, Vallès A, Lampen MH, Montenegro-Miranda PS, Keskin S, Heijink L, Evers MM, Petry H, Deventer SJV, Konstantinova P, Haan M. Spronck EA, et al. Among authors: evers mm. Brain Sci. 2021 Jan 20;11(2):129. doi: 10.3390/brainsci11020129. Brain Sci. 2021. PMID: 33498212 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: evers mm. Brain Commun. 2021 Apr 1;3(2):fcab054. doi: 10.1093/braincomms/fcab054. eCollection 2021. Brain Commun. 2021. PMID: 34704020 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: evers mm. 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.
37 results