| AAV5-miHTT gene therapy demonstrates broad distribution and strong human mutant huntingtin lowering in a Huntington’s disease minipig model MM Evers, J Miniarikova, S Juhas, A Vallès, B Bohuslavova, J Juhasova, ... Molecular Therapy 26 (9), 2163-2177, 2018 | 162 | 2018 |
| Design, characterization, and lead selection of therapeutic miRNAs targeting huntingtin for development of gene therapy for Huntington's disease J Miniarikova, I Zanella, A Huseinovic, T van der Zon, E Hanemaaijer, ... Molecular Therapy-Nucleic Acids 5, 2016 | 140 | 2016 |
| AAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington’s disease J Miniarikova, V Zimmer, R Martier, CC Brouwers, C Pythoud, K Richetin, ... Gene therapy 24 (10), 630-639, 2017 | 100 | 2017 |
| Targeting RNA-mediated toxicity in C9orf72 ALS and/or FTD by RNAi-based gene therapy R Martier, JM Liefhebber, A García-Osta, J Miniarikova, ... Molecular Therapy-Nucleic Acids 16, 26-37, 2019 | 84 | 2019 |
| Artificial microRNAs targeting C9orf72 can reduce accumulation of intra-nuclear transcripts in ALS and FTD patients R Martier, JM Liefhebber, J Miniarikova, T van der Zon, J Snapper, ... Molecular Therapy-Nucleic Acids 14, 593-608, 2019 | 65 | 2019 |
| Translation of microRNA-based huntingtin-lowering therapies from preclinical studies to the clinic J Miniarikova, MM Evers, P Konstantinova Molecular Therapy 26 (4), 947-962, 2018 | 48 | 2018 |
| Preclinical evaluation of a lentiviral vector for Huntingtin silencing K Cambon, V Zimmer, S Martineau, MC Gaillard, M Jarrige, A Bugi, ... Molecular Therapy Methods & Clinical Development 5, 259-276, 2017 | 23 | 2017 |
| RNAI induced huntingtin gene suppression PS Konstantinova, J Miniariková US Patent 10,174,321, 2019 | 14 | 2019 |
| Secreted therapeutics: monitoring durability of microRNA-based gene therapies in the central nervous system M Sogorb-Gonzalez, C Vendrell-Tornero, J Snapper, A Stam, S Keskin, ... Brain Communications 3 (2), fcab054, 2021 | 6 | 2021 |
| Transfer of therapeutic miRNAs within extracellular vesicles secreted from Huntington’s disease iPSC-derived neurons (4548) M Sogorb-Gonzalez, J Miniarikova, A Valles-sanchez, S van Deventer, ... Neurology 94 (15 Supplement), 2020 | 2 | 2020 |
| RNAi induced huntingtin gene suppression PS Konstantinova, J Miniariková US Patent 11,371,044, 2022 | 1 | 2022 |
| RNAi induced huntingtin gene suppression PS Konstantinova, J Miniariková US Patent 10,767,180, 2020 | 1 | 2020 |
| Secreted Therapeutics: Monitoring Durability of microRNA-Based Gene Therapies in Huntington's Disease C Vendrell, J Snapper, A Stam, J Miniarikova, EA Spronck, M de Haan, ... NEUROTHERAPEUTICS 17 (SUPPL 1), 20-20, 2020 | | 2020 |
| Secreted Therapeutics: Monitoring Durability of microRNA-Based Gene Therapies in Huntington's Disease M Sogorb-Gonzalez, C Vendrell, J Snapper, A Stam, J Miniarikova, ... MOLECULAR THERAPY 28 (4), 115-115, 2020 | | 2020 |
| Transfer of Therapeutic miRNAs within Extracellular Vesicles Secreted from Huntington's Disease iPSC-Derived Neurons M Sogorb-Gonzalez, J Miniarikova, A Valles, S van Deventer, ... MOLECULAR THERAPY 27 (4), 114-114, 2019 | | 2019 |
| I04 AAV5-MIHTT gene therapy demonstrates broad distribution and strong human mutant huntingtin lowering in a huntington disease minipig model J Klima, MM Evers, J Miniarikova, S Juhas, A Vallès, B Bohuslavova, ... Journal of Neurology, Neurosurgery and Psychiatry 89 (Suppl 1), A89, 2018 | | 2018 |
| MicroRNA-based gene therapy for Huntington's disease: Silencing the villain J Miniariková Mol. Ther 26, 947-962, 2018 | | 2018 |
| AAV5-miHTT gene therapy demonstrates broad vector distribution and strong mutant huntingtin lowering in a Huntington’s disease minipig model M Evers, J Miniarikova, S Juhas, A Valles-Sanchez, B Bohuslavova, ... MOLECULAR THERAPY 25 (5), 247-247, 2017 | | 2017 |
| Sustained and strong HTT silencing by AAV5-miHTT as therapy for Huntington disease M Evers, J Miniarikova, B Blits, L Spronck, C Brouwers, C Richard, ... HUMAN GENE THERAPY 27 (11), A126-A126, 2016 | | 2016 |
| SUSTAINED AND STRONG HTT SILENCING BY AAV5-MIHTT AS THERAPY FOR HUNTINGTON'S DISEASE M Evers, J Miniarikova, B Blits, L Spronck, C Brouwers, N Deglon, J Motlik, ... JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY 87, A91-A91, 2016 | | 2016 |
