| 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 | 146 | 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 | 105 | 2017 |
| Activation-induced cytidine deaminase induces reproducible DNA breaks at many non-Ig Loci in activated B cells O Staszewski, RE Baker, AJ Ucher, R Martier, J Stavnezer, JEJ Guikema Molecular cell 41 (2), 232-242, 2011 | 104 | 2011 |
| 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 | 89 | 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 | 67 | 2019 |
| Gene therapy for neurodegenerative diseases: slowing down the ticking clock R Martier, P Konstantinova Frontiers in Neuroscience 14, 580179, 2020 | 58 | 2020 |
| AAV5-miHTT lowers huntingtin mRNA and protein without off-target effects in patient-derived neuronal cultures and astrocytes S Keskin, CC Brouwers, M Sogorb-Gonzalez, R Martier, JA Depla, ... Molecular Therapy-Methods & Clinical Development 15, 275-284, 2019 | 53 | 2019 |
| Development of an AAV-based microRNA gene therapy to treat Machado-Joseph disease R Martier, M Sogorb-Gonzalez, J Stricker-Shaver, J Hübener-Schmid, ... Molecular Therapy-Methods & Clinical Development 15, 343-358, 2019 | 52 | 2019 |
| Transduction patterns in the CNS following various routes of AAV-5-mediated gene delivery KL Pietersz, RM Martier, MS Baatje, JM Liefhebber, CC Brouwers, ... Gene therapy 28 (7), 435-446, 2021 | 27 | 2021 |
| Therapeutic expression of hairpins targeting apolipoprotein B100 induces phenotypic and transcriptome changes in murine liver P Maczuga, J Verheij, C Van Der Loos, R Van Logtenstein, G Hooijer, ... Gene therapy 21 (1), 60-70, 2014 | 13 | 2014 |
| In-depth characterization of a mifepristone-regulated expression system for AAV5-mediated gene therapy in the liver JM Liefhebber, R Martier, T Van der Zon, S Keskin, A Huseinovic, ... Molecular Therapy Methods & Clinical Development 13, 512-525, 2019 | 7 | 2019 |
| Depla S Keskin, CC Brouwers, M Sogorb Gonzalez, R Martier Valles,.,… E ers, MM, 275-284, 2019 | 1 | 2019 |
| Rnai induced reduction of ataxin-3 for the treatment of spinocerebellar ataxia type 3 MM Evers, PS Konstantinova, RM Martier US Patent App. 17/319,546, 2022 | | 2022 |
| Rnai induced c9orf72 suppression for the treatment of als/ftd RM Martier, PS Konstantinova US Patent App. 17/196,531, 2021 | | 2021 |
| RNAi induced reduction of ataxin-3 for the treatment of Spinocerebellar ataxia type 3 MM Evers, PS Konstantinova, RM Martier US Patent App. 16/684,028, 2020 | | 2020 |
| Therapeutic RNAi-based gene therapy for neurodegenerative disorders: slowing down the ticking clock RM Martier Leiden University, 2020 | | 2020 |
| Baat e, MS, Liefhebber KL Pietersz, RM Martier M., Brouwers, CC, Pouw, S. M.,… Blits, B, 435-446, 2020 | | 2020 |
| A novel AAV-based miQURE gene therapy for SCA3 L Toonen, RJ Nobre, ME Haas, J Stricker-Shaver, J Hübener-Schmid, ... HUMAN GENE THERAPY 30 (11), A91-A92, 2019 | | 2019 |
| Development of an AAV-based microRNA gene therapy for treating spinocerebellar ataxia type 3 M Evers, R Martier, J Stricker-Shaver, J Hubener-Schmid, S Keskin, ... NEUROLOGY 93 (5), E531-E531, 2019 | | 2019 |
| I06 Efficacy and safety of AAV5-MIHTT in hd patient-derived neurons M Evers, S Keskin, C Brouwers, M Sogorb-Gonzalez, R Martier, J Depla, ... Journal of Neurology, Neurosurgery & Psychiatry 89 (Suppl 1), A90-A90, 2018 | | 2018 |
