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Most cited papers from Ionis Pharmaceuticals (United States)

What are the most cited papers from Ionis Pharmaceuticals (United States)?

The most cited paper from Ionis Pharmaceuticals (United States) is "Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy" (Richard S. Finkel et al., 2017), with 2,357 citations. Next come "The Nuclear-Retained Noncoding RNA MALAT1 Regulates Alternative Splicing by Modulating SR Splicing Factor Phosphorylation" (2,272) and "PNA hybridizes to complementary oligonucleotides obeying the Watson–Crick hydrogen-bonding rules" (2,092).

Showing 50 of 60 papers. Every paper with more than 551 citations is listed.

Most cited papers, sortable by citations and release date
#Paper
1 Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy Richard S. Finkel et al. · New England Journal of Medicine Open access Nov 2017 2,357
2 The Nuclear-Retained Noncoding RNA MALAT1 Regulates Alternative Splicing by Modulating SR Splicing Factor Phosphorylation Vidisha Tripathi, Jonathan D. Ellis, Zhen Shen et al. · Molecular Cell Open access 27 Aug 2010 2,272
3 PNA hybridizes to complementary oligonucleotides obeying the Watson–Crick hydrogen-bonding rules Michael Egholm, Ole Buchardt, Leif Christensen et al. · Nature Oct 1993 2,092
4 miR-122 regulation of lipid metabolism revealed by in vivo antisense targeting Christine Esau, Scott P. Davis, Susan Murray et al. · Cell Metabolism Open access Feb 2006 2,088
5 PTEN activation contributes to tumor inhibition by trastuzumab, and loss of PTEN predicts trastuzumab resistance in patients Yoichi Nagata, Keng‐Hsueh Lan, Xiaoyan Zhou et al. · Cancer Cell Open access Aug 2004 1,878
6 ElliPro: a new structure-based tool for the prediction of antibody epitopes Julia V. Ponomarenko, Huynh‐Hoa Bui, Wei Li et al. · BMC Bioinformatics Open access Dec 2008 1,787
7 Robust transcriptome-wide discovery of RNA-binding protein binding sites with enhanced CLIP (eCLIP) Eric L. Van Nostrand, Gabriel A. Pratt, Alexander A. Shishkin et al. · Nature Methods Open access 28 Mar 2016 1,619
8 The Noncoding RNA MALAT1 Is a Critical Regulator of the Metastasis Phenotype of Lung Cancer Cells Tony Gutschner, Monika Hämmerle, Moritz F. Eissmann et al. · Cancer Research Open access 15 Dec 2012 1,597
9 Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy Eugenio Maria Mercuri et al. · New England Journal of Medicine Open access 14 Feb 2018 1,466
10 Long pre-mRNA depletion and RNA missplicing contribute to neuronal vulnerability from loss of TDP-43 Magdalini Polymenidou, Clotilde Lagier‐Tourenne, Kasey R. Hutt et al. · Nature Neuroscience Open access 27 Feb 2011 1,398
11 RNA Targeting Therapeutics: Molecular Mechanisms of Antisense Oligonucleotides as a Therapeutic Platform C. Frank Bennett, Eric E. Swayze · The Annual Review of Pharmacology and Toxicology Review 8 Jan 2010 1,384
12 Metformin suppresses gluconeogenesis by inhibiting mitochondrial glycerophosphate dehydrogenase Anila K. Madiraju, Derek M. Erion, Yasmeen Rahimi et al. · Nature Open access 21 May 2014 1,311
13 Constitutive Stat3 activity up-regulates VEGF expression and tumor angiogenesis Guilian Niu, Kenneth L. Wright, Mei Huang et al. · Oncogene 21 Mar 2002 1,218
14 Trimethylamine-N-Oxide, a Metabolite Associated with Atherosclerosis, Exhibits Complex Genetic and Dietary Regulation Brian J. Bennett, Thomas Q. de Aguiar Vallim, Zeneng Wang et al. · Cell Metabolism Open access 2013 1,084
15 Lipoprotein(a) Reduction in Persons with Cardiovascular Disease Sotirios Tsimikas, Ewa Karwatowska‐Prokopczuk, Ioanna Gouni‐Berthold et al. · New England Journal of Medicine Open access 2020 1,075
16 Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study Richard S. Finkel, Claudia A. Chiriboga, Jiri Vajsar et al. · The Lancet Dec 2016 1,043
17 MicroRNA-143 Regulates Adipocyte Differentiation Christine Esau, Xiaolin Kang, Eigen R. Peralta et al. · Journal of Biological Chemistry Open access 26 Oct 2004 1,029
18 Inhibiting triglyceride synthesis improves hepatic steatosis but exacerbates liver damage and fibrosis in obese mice with nonalcoholic steatohepatitis Kanji Yamaguchi, Liu Yang, Shannon Jones McCall et al. · Hepatology Open access 3 May 2007 1,022
19 CD36 coordinates NLRP3 inflammasome activation by facilitating intracellular nucleation of soluble ligands into particulate ligands in sterile inflammation Frederick J. Sheedy, Alena Grebe, Katey J. Rayner et al. · Nature Immunology Open access 30 Jun 2013 935
20 Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trial Frederick Johan Raal, Raul Dias Santos, Dirk Jacobus Blom et al. · The Lancet Mar 2010 921
21 RNA Toxicity from the ALS/FTD C9ORF72 Expansion Is Mitigated by Antisense Intervention Christopher J. Donnelly, Ping‐Wu Zhang, Jacqueline T. Pham et al. · Neuron Open access Oct 2013 895
22 Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides Richard S. Geary, Daniel A. Norris, Rosie Z. Yu et al. · Advanced Drug Delivery Reviews ReviewOpen access 7 Feb 2015 863
23 RNA-Targeted Therapeutics Stanley T. Crooke, Joseph Lee Witztum, C. Frank Bennett et al. · Cell Metabolism Open access Apr 2018 834
24 Management of Acute Myocarditis and Chronic Inflammatory Cardiomyopathy Enrico Ammirati, Maria Frigerio, ERIC D. ADLER et al. · Circulation Heart Failure Open access Nov 2020 809
25 Long Noncoding RNA MALAT1 Controls Cell Cycle Progression by Regulating the Expression of Oncogenic Transcription Factor B-MYB Vidisha Tripathi, Zhen Shen, Arindam Chakraborty et al. · PLoS Genetics Open access 21 Mar 2013 789
26 Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis Holly B. Kordasiewicz, Lisa M. Stanek, Edward V. Wancewicz et al. · Neuron Open access Jun 2012 787
27 Evaluation of 2‘-modified oligonucleotides containing 2‘-deoxy gaps as antisense inhibitors of gene expression Brett P. Monia, Elena A. Lesnik, C. Gonzalez et al. · Journal of Biological Chemistry Open access Jul 1993 780
28 Divergent roles of ALS-linked proteins FUS/TLS and TDP-43 intersect in processing long pre-mRNAs Clotilde Lagier‐Tourenne et al. · Nature Neuroscience Open access 30 Sep 2012 764
29 Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model Yimin Hua, Kentaro Sahashi, Frank Rigo et al. · Nature Open access Oct 2011 760
30 Antisense technology: an overview and prospectus Stanley T. Crooke, Brenda F. Baker, Rosanne M. Crooke et al. · Nature Reviews Drug Discovery Review 24 Mar 2021 759
31 Antisense oligonucleotides targeting apolipoprotein(a) in people with raised lipoprotein(a): two randomised, double-blind, placebo-controlled, dose-ranging trials Nicholas J. Viney, Julian C. van Capelleveen, Richard S. Geary et al. · The Lancet 21 Sep 2016 759
32 3′ End Processing of a Long Nuclear-Retained Noncoding RNA Yields a tRNA-like Cytoplasmic RNA Jeremy E. Wilusz, Susan M. Freier, David L. Spector · Cell Open access Nov 2008 737
33 Regulating Gene Expression through RNA Nuclear Retention Kannanganattu V. Prasanth, Supriya G. Prasanth, Zhenyu Xuan et al. · Cell Open access Oct 2005 717
34 Patterns of Variant Polyadenylation Signal Usage in Human Genes Emmanuel Beaudoing, Susan M. Freier, Jacqueline R. Wyatt et al. · Genome Research Open access Jul 2000 705
35 Targeting RNA Foci in iPSC-Derived Motor Neurons from ALS Patients with a C9ORF72 Repeat Expansion Dhruv Sareen, Jacqueline G. O’Rourke, Pratap Meera et al. · Science Translational Medicine Open access 23 Oct 2013 697
36 The ups and downs of nucleic acid duplex stability: structure-stability studies on chemically-modified DNA:RNA duplexes Sandra Freier · Nucleic Acids Research Open access 15 Nov 1997 692
37 Premature polyadenylation-mediated loss of stathmin-2 is a hallmark of TDP-43-dependent neurodegeneration Ze’ev Melamed, Jone López‐Erauskin, Michael W. Baughn et al. · Nature Neuroscience Open access 8 Jan 2019 689
38 Volanesorsen and Triglyceride Levels in Familial Chylomicronemia Syndrome Joseph Lee Witztum, Daniel Gaudet, Steven D. Freedman et al. · New England Journal of Medicine Open access 7 Aug 2019 687
39 Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model Yimin Hua, Kentaro Sahashi, Gene Hung et al. · Genes & Development Open access 12 Jul 2010 677
40 Inhibition of STAT3 signaling leads to apoptosis of leukemic large granular lymphocytes and decreased Mcl-1 expression Pearlie K. Epling‐Burnette, Jin Hong Liu, Robyn Catlett-Falcone et al. · Journal of Clinical Investigation Open access Feb 2001 647
41 Factor XI Antisense Oligonucleotide for Prevention of Venous Thrombosis Harry Roger Buller, Claudette Bethune, Sanjay Bhanot et al. · New England Journal of Medicine Open access 7 Dec 2014 632
42 Results from a phase 1 study of nusinersen (ISIS-SMN Rx ) in children with spinal muscular atrophy Claudia A. Chiriboga, Kathryn J. Swoboda, Basil T. Darras et al. · Neurology Open access 11 Feb 2016 618
43 An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study Timothy M. Miller, Alan Pestronk, William David et al. · The Lancet Neurology Open access 28 Mar 2013 618
44 Targeted delivery of antisense oligonucleotides to hepatocytes using triantennaryN-acetyl galactosamine improves potency 10-fold in mice Thazha P. Prakash, Mark J. Graham, Jinghua Yu et al. · Nucleic Acids Research Open access 3 Jul 2014 618
45 Cationic lipids enhance cellular uptake and activity of phosphorothioate antisense oligonucleotides. C. Frank Bennett, M Y Chiang, Huai En Huang Chan et al. · Molecular Pharmacology Jun 1992 618
46 The lncRNA Malat1 Is Dispensable for Mouse Development but Its Transcription Plays a cis-Regulatory Role in the Adult Bin Zhang, Gayatri Arun, Yuntao S. Mao et al. · Cell Reports Open access 28 Jun 2012 615
47 Pleiotropic cell-division defects and apoptosis induced by interference with survivin function Fengzhi Li, Elizabeth J. Ackermann, C. Frank Bennett et al. · Nature Cell Biology 27 Oct 1999 605
48 Therapeutic Antisense Oligonucleotides Are Coming of Age C. Frank Bennett · Annual Review of Medicine ReviewOpen access 27 Jan 2019 603
49 Therapeutic reduction of ataxin-2 extends lifespan and reduces pathology in TDP-43 mice Lindsay A. Becker, Brenda Huang, Gregor Bieri et al. · Nature Open access 11 Apr 2017 598
50 Cardiovascular and Metabolic Effects of ANGPTL3 Antisense Oligonucleotides Mark J. Graham, Richard G. Lee, Teresa A. Brandt et al. · New England Journal of Medicine Open access 24 May 2017 587

Citations are OpenAlex's own counts, from its 2026-09-23 snapshot, so the ranking can differ from Google Scholar or Web of Science. A paper is listed for an institution when any of its authors gave it as an affiliation; retracted papers and papers whose citation count cannot be traced to citing works are left out, and a preprint and the article it became are shown once. Release dates are OpenAlex's: where a source gave only a month or a year, that is what is shown. How the lists are chosen.