Search Results - "FITZGERALD, KEVIN"
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The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs
Published in Nature nanotechnology (01-12-2019)“…The regulatory approval of Onpattro, a lipid nanoparticle-based short interfering RNA drug for the treatment of polyneuropathies induced by hereditary…”
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2
A Highly Durable RNAi Therapeutic Inhibitor of PCSK9
Published in The New England journal of medicine (05-01-2017)“…In a phase 1 trial, healthy volunteers were assigned to an RNAi therapeutic inhibitor of PCSK9 or placebo. Single doses of 300 mg or more reduced LDL…”
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3
In Vivo Silencing of the Transcription Factor IRF5 Reprograms the Macrophage Phenotype and Improves Infarct Healing
Published in Journal of the American College of Cardiology (22-04-2014)“…Objectives The aim of this study was to test whether silencing of the transcription factor interferon regulatory factor 5 (IRF5) in cardiac macrophages…”
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Acetyl CoA Carboxylase Inhibition Reduces Hepatic Steatosis but Elevates Plasma Triglycerides in Mice and Humans: A Bedside to Bench Investigation
Published in Cell metabolism (01-08-2017)“…Inhibiting lipogenesis prevents hepatic steatosis in rodents with insulin resistance. To determine if reducing lipogenesis functions similarly in humans, we…”
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An Investigational RNAi Therapeutic Targeting Glycolate Oxidase Reduces Oxalate Production in Models of Primary Hyperoxaluria
Published in Journal of the American Society of Nephrology (01-02-2017)“…Primary hyperoxaluria type 1 (PH1), an inherited rare disease of glyoxylate metabolism, arises from mutations in the enzyme alanine-glyoxylate…”
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6
Proliferation and Recruitment Contribute to Myocardial Macrophage Expansion in Chronic Heart Failure
Published in Circulation research (16-09-2016)“…RATIONALE:Macrophages reside in the healthy myocardium, participate in ischemic heart disease and modulate myocardial infarction (MI) healing. Their origin and…”
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7
Image-based analysis of lipid nanoparticle–mediated siRNA delivery, intracellular trafficking and endosomal escape
Published in Nature biotechnology (01-07-2013)“…Detailed analyses of siRNA delivery by lipid nanoparticles reveal major pathways of siRNA internalization and endosomal escape. Delivery of short interfering…”
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Divergent effects of glucose and fructose on hepatic lipogenesis and insulin signaling
Published in The Journal of clinical investigation (01-11-2017)“…Overconsumption of high-fat diet (HFD) and sugar-sweetened beverages are risk factors for developing obesity, insulin resistance, and fatty liver disease. Here…”
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Multivalent N‑Acetylgalactosamine-Conjugated siRNA Localizes in Hepatocytes and Elicits Robust RNAi-Mediated Gene Silencing
Published in Journal of the American Chemical Society (10-12-2014)“…Conjugation of small interfering RNA (siRNA) to an asialoglycoprotein receptor ligand derived from N-acetylgalactosamine (GalNAc) facilitates targeted delivery…”
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10
Dietary Sugars Alter Hepatic Fatty Acid Oxidation via Transcriptional and Post-translational Modifications of Mitochondrial Proteins
Published in Cell metabolism (01-10-2019)“…Dietary sugars, fructose and glucose, promote hepatic de novo lipogenesis and modify the effects of a high-fat diet (HFD) on the development of insulin…”
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11
Suburbanization, estrogen contamination, and sex ratio in wild amphibian populations
Published in Proceedings of the National Academy of Sciences - PNAS (22-09-2015)“…Research on endocrine disruption in frog populations, such as shifts in sex ratios and feminization of males, has predominantly focused on agricultural…”
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12
Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria
Published in The New England journal of medicine (07-02-2019)“…Acute intermittent porphyria results in excess activity of ALA synthase and overproduction of neurotoxic metabolites that cause attacks of severe abdominal…”
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13
Impact of enhanced metabolic stability on pharmacokinetics and pharmacodynamics of GalNAc-siRNA conjugates
Published in Nucleic acids research (02-11-2017)“…Covalent attachment of a synthetic triantennary N-acetylagalactosamine (GalNAc) ligand to chemically modified siRNA has enabled asialoglycoprotein…”
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14
Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial
Published in The Lancet (British edition) (04-01-2014)“…Summary Background Proprotein convertase subtilisin/kexin type 9 (PCSK9) binds to LDL receptors, leading to their degradation. Genetics studies have shown that…”
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15
Lipopeptide nanoparticles for potent and selective siRNA delivery in rodents and nonhuman primates
Published in Proceedings of the National Academy of Sciences - PNAS (18-03-2014)“…siRNA therapeutics have promise for the treatment of a wide range of genetic disorders. Motivated by lipoproteins, we report lipopeptide nanoparticles as…”
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16
Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis
Published in The New England journal of medicine (29-08-2013)“…Transthyretin amyloidosis is largely caused by synthesis of mutant transthyretin in the liver and deposition of transthyretin in other organs. A therapeutic…”
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The Scap/SREBP Pathway Is Essential for Developing Diabetic Fatty Liver and Carbohydrate-Induced Hypertriglyceridemia in Animals
Published in Cell metabolism (08-02-2012)“…Insulin resistance leads to hypertriglyceridemia and hepatic steatosis and is associated with increased SREBP-1c, a transcription factor that activates fatty…”
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18
Monocyte-Directed RNAi Targeting CCR2 Improves Infarct Healing in Atherosclerosis-Prone Mice
Published in Circulation (New York, N.Y.) (21-05-2013)“…BACKGROUND—Exaggerated and prolonged inflammation after myocardial infarction (MI) accelerates left ventricular remodeling. Inflammatory pathways may present a…”
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From noncoding variant to phenotype via SORT1 at the 1p13 cholesterol locus
Published in Nature (London) (05-08-2010)“…Recent genome-wide association studies (GWASs) have identified a locus on chromosome 1p13 strongly associated with both plasma low-density lipoprotein…”
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Ablation in Mice of the mTORC Components raptor, rictor, or mLST8 Reveals that mTORC2 Is Required for Signaling to Akt-FOXO and PKCα, but Not S6K1
Published in Developmental cell (01-12-2006)“…The mTOR kinase controls cell growth, proliferation, and survival through two distinct multiprotein complexes, mTORC1 and mTORC2. mTOR and mLST8 are in both…”
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