| Robust transcriptome-wide discovery of RNA-binding protein binding sites with enhanced CLIP (eCLIP) EL Van Nostrand, GA Pratt, AA Shishkin, C Gelboin-Burkhart, MY Fang, ... Nature methods 13 (6), 508-514, 2016 | 1213 | 2016 |
| Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study RS Finkel, CA Chiriboga, J Vajsar, JW Day, J Montes, DC De Vivo, ... The Lancet 388 (10063), 3017-3026, 2016 | 1014 | 2016 |
| RNA toxicity from the ALS/FTD C9ORF72 expansion is mitigated by antisense intervention CJ Donnelly, PW Zhang, JT Pham, AR Haeusler, NA Mistry, S Vidensky, ... Neuron 80 (2), 415-428, 2013 | 1003 | 2013 |
| Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model Y Hua, K Sahashi, F Rigo, G Hung, G Horev, CF Bennett, AR Krainer Nature 478 (7367), 123-126, 2011 | 765 | 2011 |
| Targeting RNA foci in iPSC-derived motor neurons from ALS patients with a C9ORF72 repeat expansion D Sareen, JG O’Rourke, P Meera, A Muhammad, S Grant, M Simpkinson, ... Science translational medicine 5 (208), 208ra149-208ra149, 2013 | 757 | 2013 |
| Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model Y Hua, K Sahashi, G Hung, F Rigo, MA Passini, CF Bennett, AR Krainer Genes & development 24 (15), 1634-1644, 2010 | 726 | 2010 |
| Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration C Lagier-Tourenne, M Baughn, F Rigo, S Sun, P Liu, HR Li, J Jiang, ... Proceedings of the National Academy of Sciences 110 (47), E4530-E4539, 2013 | 657 | 2013 |
| Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy MA Passini, J Bu, AM Richards, C Kinnecom, SP Sardi, LM Stanek, Y Hua, ... Science translational medicine 3 (72), 72ra18-72ra18, 2011 | 560 | 2011 |
| Gain of toxicity from ALS/FTD-linked repeat expansions in C9ORF72 is alleviated by antisense oligonucleotides targeting GGGGCC-containing RNAs J Jiang, Q Zhu, TF Gendron, S Saberi, M McAlonis-Downes, A Seelman, ... Neuron 90 (3), 535-550, 2016 | 556 | 2016 |
| Therapeutic reduction of ataxin-2 extends lifespan and reduces pathology in TDP-43 mice LA Becker, B Huang, G Bieri, R Ma, DA Knowles, P Jafar-Nejad, ... Nature 544 (7650), 367-371, 2017 | 515 | 2017 |
| Towards a therapy for Angelman syndrome by targeting a long non-coding RNA L Meng, AJ Ward, S Chun, CF Bennett, AL Beaudet, F Rigo Nature 518 (7539), 409-412, 2015 | 505 | 2015 |
| The long non-coding RNA Gomafu is acutely regulated in response to neuronal activation and involved in schizophrenia-associated alternative splicing G Barry, JA Briggs, DP Vanichkina, EM Poth, NJ Beveridge, VS Ratnu, ... Molecular psychiatry 19 (4), 486-494, 2014 | 410 | 2014 |
| Premature polyadenylation-mediated loss of stathmin-2 is a hallmark of TDP-43-dependent neurodegeneration Z Melamed, J López-Erauskin, MW Baughn, O Zhang, K Drenner, Y Sun, ... Nature neuroscience 22 (2), 180-190, 2019 | 387 | 2019 |
| Stress granule assembly disrupts nucleocytoplasmic transport K Zhang, JG Daigle, KM Cunningham, AN Coyne, K Ruan, JC Grima, ... Cell 173 (4), 958-971. e17, 2018 | 347 | 2018 |
| Antisense reduction of tau in adult mice protects against seizures SL DeVos, DK Goncharoff, G Chen, CS Kebodeaux, K Yamada, ... Journal of Neuroscience 33 (31), 12887-12897, 2013 | 335 | 2013 |
| Antisense oligonucleotide therapy for spinocerebellar ataxia type 2 DR Scoles, P Meera, MD Schneider, S Paul, W Dansithong, KP Figueroa, ... Nature 544 (7650), 362-366, 2017 | 312 | 2017 |
| Pharmacology of a central nervous system delivered 2′-O-methoxyethyl–modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates F Rigo, SJ Chun, DA Norris, G Hung, S Lee, J Matson, RA Fey, H Gaus, ... Journal of Pharmacology and Experimental Therapeutics 350 (1), 46-55, 2014 | 273 | 2014 |
| Poly(GP) proteins are a useful pharmacodynamic marker for C9ORF72-associated amyotrophic lateral sclerosis TF Gendron, J Chew, JN Stankowski, LR Hayes, YJ Zhang, M Prudencio, ... Science translational medicine 9 (383), eaai7866, 2017 | 223 | 2017 |
| Rescue of hearing and vestibular function by antisense oligonucleotides in a mouse model of human deafness JJ Lentz, FM Jodelka, AJ Hinrich, KE McCaffrey, HE Farris, MJ Spalitta, ... Nature medicine 19 (3), 345-350, 2013 | 216 | 2013 |
| Reversal of phenotypes in MECP2 duplication mice using genetic rescue or antisense oligonucleotides Y Sztainberg, H Chen, JW Swann, S Hao, B Tang, Z Wu, J Tang, YW Wan, ... Nature 528 (7580), 123-126, 2015 | 209 | 2015 |
