| # | Title | Journal | Year | Citations |
|---|
| 1 | Lipoprotein(a) Reduction in Persons with Cardiovascular Disease | New England Journal of Medicine | 2020 | 559 |
| 2 | The human lncRNA LINC-PINT inhibits tumor cell invasion through a highly conserved sequence element | Genome Biology | 2017 | 161 |
| 3 | Neurocalcin Delta Suppression Protects against Spinal Muscular Atrophy in Humans and across Species by Restoring Impaired Endocytosis | American Journal of Human Genetics | 2017 | 156 |
| 4 | The Power of Human Protective Modifiers: PLS3 and CORO1C Unravel Impaired Endocytosis in Spinal Muscular Atrophy and Rescue SMA Phenotype | American Journal of Human Genetics | 2016 | 154 |
| 5 | c‐Jun N‐terminal kinase mediates mouse liver injury through a novel Sab (SH3BP5)‐dependent pathway leading to inactivation of intramitochondrial Src | Hepatology | 2016 | 146 |
| 6 | Oligonucleotide therapy mitigates disease in spinocerebellar ataxia type 3 mice | Annals of Neurology | 2018 | 127 |
| 7 | RIPK1 Expression Associates With Inflammation in Early Atherosclerosis in Humans and Can Be Therapeutically Silenced to Reduce NF-κB Activation and Atherogenesis in Mice | Circulation | 2021 | 102 |
| 8 | A lncRNA-SWI/SNF complex crosstalk controls transcriptional activation at specific promoter regions | Nature Communications | 2020 | 69 |
| 9 | Prosurvival long noncoding RNA PINCR regulates a subset of p53 targets in human colorectal cancer cells by binding to Matrin 3 | ELife | 2017 | 68 |
| 10 | Antisense suppression of glial fibrillary acidic protein as a treatment for Alexander disease | Annals of Neurology | 2018 | 62 |
| 11 | The role of MAP2 kinases and p38 kinase in acute murine liver injury models | Cell Death and Disease | 2017 | 53 |
| 12 | Argonaute 2-dependent Regulation of Gene Expression by Single-stranded miRNA Mimics | Molecular Therapy | 2016 | 51 |
| 13 | CHP1 reduction ameliorates spinal muscular atrophy pathology by restoring calcineurin activity and endocytosis | Brain | 2018 | 49 |
| 14 | Elucidation of the Biotransformation Pathways of a Galnac3-conjugated Antisense Oligonucleotide in Rats and Monkeys | Molecular Therapy - Nucleic Acids | 2016 | 46 |
| 15 | Loss of MLKL (Mixed Lineage Kinase Domain-Like Protein) Decreases Necrotic Core but Increases Macrophage Lipid Accumulation in Atherosclerosis | Arteriosclerosis, Thrombosis, and Vascular Biology | 2020 | 42 |
| 16 | Population Pharmacokinetics of Nusinersen in the Cerebral Spinal Fluid and Plasma of Pediatric Patients With Spinal Muscular Atrophy Following Intrathecal Administrations | Journal of Clinical Pharmacology | 2017 | 38 |
| 17 | A novel humanized mouse model of Huntington disease for preclinical development of therapeutics targeting mutant huntingtin alleles | Human Molecular Genetics | 2017 | 37 |
| 18 | Temporal variability in lipoprotein(a) levels in patients enrolled in the placebo arms of IONIS-APO(a)Rx and IONIS-APO(a)-LRx antisense oligonucleotide clinical trials | Journal of Clinical Lipidology | 2018 | 36 |
| 19 | Serine/threonine protein kinase 25 antisense oligonucleotide treatment reverses glucose intolerance, insulin resistance, and nonalcoholic fatty liver disease in mice | Hepatology Communications | 2018 | 35 |
| 20 | Pharmacokinetic and Pharmacodynamic Investigations of ION-353382, a Model Antisense Oligonucleotide: Using Alpha-2-Macroglobulin and Murinoglobulin Double-Knockout Mice | Nucleic Acid Therapeutics | 2016 | 34 |
| 21 | RIPK1 gene variants associate with obesity in humans and can be therapeutically silenced to reduce obesity in mice | Nature Metabolism | 2020 | 34 |
| 22 | A MAFG-lncRNA axis links systemic nutrient abundance to hepatic glucose metabolism | Nature Communications | 2020 | 29 |
| 23 | The Distinct and Cooperative Roles of Toll-Like Receptor 9 and Receptor for Advanced Glycation End Products in ModulatingIn VivoInflammatory Responses to Select CpG and Non-CpG Oligonucleotides | Nucleic Acid Therapeutics | 2017 | 24 |
| 24 | Hepatic Mitochondrial SAB Deletion or Knockdown Alleviates Diet‐Induced Metabolic Syndrome, Steatohepatitis, and Hepatic Fibrosis | Hepatology | 2021 | 24 |
| 25 | The approach study: a randomized, double-blind, placebo-controlled, phase 3 study of volanesorsen administered subcutaneously to patients with familial chylomicronemia syndrome (FCS) | Atherosclerosis | 2017 | 23 |
| 26 | TFEB-dependent induction of thermogenesis by the hepatocyte SLC2A inhibitor trehalose | Autophagy | 2018 | 23 |
| 27 | Double-Stranded RNA Is a Novel Molecular Target in Osteomyelitis Pathogenesis | American Journal of Pathology | 2019 | 23 |
| 28 | Assessment of the Effects of 2′-Methoxyethyl Antisense Oligonucleotides on Platelet Count in Cynomolgus Nonhuman Primates | Nucleic Acid Therapeutics | 2017 | 22 |
| 29 | Methionine adenosyltransferase 1a antisense oligonucleotides activate the liver-brown adipose tissue axis preventing obesity and associated hepatosteatosis | Nature Communications | 2022 | 22 |
| 30 | Hepatocyte ALOXE3 is induced during adaptive fasting and enhances insulin sensitivity by activating hepatic PPARγ | JCI Insight | 2018 | 21 |
| 31 | Hereditary transthyretin amyloidosis: baseline characteristics of patients in the NEURO-TTR trial | Amyloid: the International Journal of Experimental and Clinical Investigation: the Official Journal of the International Society of Amyloidosis | 2018 | 20 |
| 32 | Role for Cela1 in Postnatal Lung Remodeling and Alpha-1 Antitrypsin–Deficient Emphysema | American Journal of Respiratory Cell and Molecular Biology | 2018 | 19 |
| 33 | Structural Determinants for the Interactions of Chemically Modified Nucleic Acids with the Stabilin-2 Clearance Receptor | Biochemistry | 2018 | 17 |
| 34 | Astrocytic 4R tau expression drives astrocyte reactivity and dysfunction | JCI Insight | 2022 | 15 |
| 35 | Design, synthesis, and duplex-stabilizing properties of conformationally constrained tricyclic analogues of LNA | Organic and Biomolecular Chemistry | 2016 | 14 |
| 36 | Limited role of kininogen in the host response during gram-negative pneumonia-derived sepsis | American Journal of Physiology - Lung Cellular and Molecular Physiology | 2018 | 12 |
| 37 | Population pharmacokinetics and pharmacodynamics of IONIS-GCGRRx, an antisense oligonucleotide for type 2 diabetes mellitus: a red blood cell lifespan model | Journal of Pharmacokinetics and Pharmacodynamics | 2017 | 11 |
| 38 | Evaluation of potential effects of Plastin 3 overexpression and low-dose SMN-antisense oligonucleotides on putative biomarkers in spinal muscular atrophy mice | PLoS ONE | 2018 | 11 |
| 39 | The 6th International Lafora Epilepsy Workshop: Advances in the search for a cure | Epilepsy and Behavior | 2021 | 11 |
| 40 | Heparin-binding EGF-like growth factor (HB-EGF) antisense oligonucleotide protected against hyperlipidemia-associated atherosclerosis | Nutrition, Metabolism and Cardiovascular Diseases | 2019 | 10 |
| 41 | Specific targeting of the KRAS mutational landscape in myeloma as a tool to unveil the elicited antitumor activity | Blood | 2021 | 10 |
| 42 | Evaluation of Phosphorus and Non-Phosphorus Neutral Oligonucleotide Backbones for Enhancing Therapeutic Index of Gapmer Antisense Oligonucleotides | Nucleic Acid Therapeutics | 2022 | 10 |
| 43 | Exploring the characteristics and most bothersome symptoms in MECP2 duplication syndrome to pave the path toward developing parent‐oriented outcome measures | Molecular Genetics & Genomic Medicine | 2022 | 8 |
| 44 | Mouse genome-wide association studies and systems genetics uncover the genetic architecture associated with hepatic pharmacokinetic and pharmacodynamic properties of a constrained ethyl antisense oligonucleotide targeting Malat1 | PLoS Genetics | 2018 | 7 |
| 45 | Transmembrane Protease, Serine 6 (TMPRSS6) Antisense Oligonucleotide (IONIS-TMPRSS6-LRX) Reduces Plasma Iron Levels of Healthy Volunteers in a Phase 1 Clinical Study | Blood | 2018 | 7 |
| 46 | Reducing YAP expression in Pkd1 mutant mice does not improve the cystic phenotype | Journal of Cellular and Molecular Medicine | 2020 | 5 |
| 47 | Hybrid Mouse Diversity Panel Identifies Genetic Architecture Associated with the Acute Antisense Oligonucleotide-Mediated Inflammatory Response to a 2′-O-Methoxyethyl Antisense Oligonucleotide | Nucleic Acid Therapeutics | 2019 | 4 |
| 48 | Prothrombin, alone or in complex concentrates or plasma, reduces bleeding in a mouse model of blood exchange-induced coagulopathy | Scientific Reports | 2019 | 3 |
| 49 | Abstract 13307: An Oral Antisense Oligonucleotide for PCSK9 Inhibition in Humans | Circulation | 2020 | 3 |
| 50 | Knockdown of Z Mutant Alpha-1 Antitrypsin In Vivo Using Modified DNA Antisense Oligonucleotides | Methods in Molecular Biology | 2017 | 2 |
