Therapeutic RNA-silencing oligonucleotides in metabolic diseases

Inclisiran: First Approval

(2021) Yvette N. Lamb   DRUGS

Exome-Wide Association Study on Alanine Aminotransferase Identifies Sequence Variants in the GPAM and APOE Associated With Fatty Liver Disease

(2021) Oveis Jamialahmadi et al.   GASTROENTEROLOGY

Pharmacologic inhibition of Ketohexokinase prevents fructose-induced metabolic dysfunction

(2021) Jemy A. Gutierrez et al.   Molecular Metabolism

Loss-of-function HSD17B13 variants, non-alcoholic steatohepatitis and adverse liver outcomes: results from a multi-ethnic Asian cohort

(2021) Yi-Wen Ting et al.   Clinical and Molecular Hepatology

Phase 1/2 Study of Lumasiran for Treatment of Primary Hyperoxaluria Type 1

(2021) Yaacov Frishberg et al.   Clinical Journal of the American Society of Nephrology

SAFETY, PHARMACODYNAMICS, AND BLOOD PRESSURE EFFECTS OF ALN-AGT, AN RNA INTERFERENCE THERAPEUTIC TARGETING ANGIOTENSINOGEN, IN A RANDOMIZED SINGLE ASCENDING DOSE STUDY OF HYPERTENSIVE ADULTS

(2021) Stephen A. Huang et al.   JOURNAL OF HYPERTENSION

Dysregulation of homocysteine homeostasis in acute intermittent porphyria patients receiving heme arginate or givosiran

(2021) Jordi To‐Figueras et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Advances in the Design of (Nano)Formulations for Delivery of Antisense Oligonucleotides and Small Interfering RNA: Focus on the Central Nervous System

(2021) Monique C. P. Mendonça et al.   MOLECULAR PHARMACEUTICS

Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1

(2021) Sander F. Garrelfs et al.   NEW ENGLAND JOURNAL OF MEDICINE

An oral antisense oligonucleotide for PCSK9 inhibition

(2021) Peter Gennemark et al.   Science Translational Medicine

Efficacy and safety of volanesorsen in patients with multifactorial chylomicronaemia (COMPASS): a multicentre, double-blind, randomised, placebo-controlled, phase 3 trial

(2021) Ioanna Gouni-Berthold et al.   Lancet Diabetes & Endocrinology

The Challenges and Strategies of Antisense Oligonucleotide Drug Delivery

(2021) Maria Gagliardi et al.   Biomedicines

Fructose and fructose kinase in cancer and other pathologies

(2021) Hongfei Jiang et al.   Journal of Genetics and Genomics

Towards next generation antisense oligonucleotides: mesylphosphoramidate modification improves therapeutic index and duration of effect of gapmer antisense oligonucleotides

(2021) Brooke A Anderson et al.   NUCLEIC ACIDS RESEARCH

Sequencing of 640,000 exomes identifies GPR75 variants associated with protection from obesity

(2021) Parsa Akbari et al.   SCIENCE

Dual Glycolate Oxidase/Lactate Dehydrogenase A Inhibitors for Primary Hyperoxaluria

(2021) Jinyue Ding et al.   ACS Medicinal Chemistry Letters

Toxicological profile of lipid-based nanostructures: are they considered as completely safe nanocarriers?

(2020) Asaad Azarnezhad et al.   CRITICAL REVIEWS IN TOXICOLOGY

Givosiran: First Approval

(2020) Lesley J. Scott   DRUGS

Effect of Alirocumab on Lipoprotein(a) and Cardiovascular Risk After Acute Coronary Syndrome

(2020) Vera A. Bittner et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Mechanisms of Cardiorenal Effects of Sodium-Glucose Cotransporter 2 Inhibitors

(2020) Thomas A. Zelniker et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Lipoprotein(a) Reduction in Persons with Cardiovascular Disease

(2020) Sotirios Tsimikas et al.   NEW ENGLAND JOURNAL OF MEDICINE

Lipoprotein(a) plasma levels are not associated with survival after acute coronary syndromes: An observational cohort study

(2020) Christian Roth et al.   PLoS One

Hyperhomocysteinemia in patients with acute porphyrias: A potentially dangerous metabolic crossroad?

(2020) Paolo Ventura et al.   European Journal of Internal Medicine

Genome-wide association study of non-alcoholic fatty liver and steatohepatitis in a histologically-characterised cohort

(2020) Quentin M. Anstee et al.   JOURNAL OF HEPATOLOGY

Fructose Production and Metabolism in the Kidney

(2020) Takahiko Nakagawa et al.   JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY

Two Phase 3 Trials of Inclisiran in Patients with Elevated LDL Cholesterol

(2020) Kausik K. Ray et al.   NEW ENGLAND JOURNAL OF MEDICINE

Inclisiran for the Treatment of Heterozygous Familial Hypercholesterolemia

(2020) Frederick J. Raal et al.   NEW ENGLAND JOURNAL OF MEDICINE

A missense variant in Mitochondrial Amidoxime Reducing Component 1 gene and protection against liver disease

(2020) Connor A. Emdin et al.   PLoS Genetics

Characterising a healthy adult with a rare HAO1 knockout to support a therapeutic strategy for primary hyperoxaluria

(2020) Tracy L McGregor et al.   eLife

Recent advances in siRNA delivery mediated by lipid-based nanoparticles

(2020) Sei Yonezawa et al.   ADVANCED DRUG DELIVERY REVIEWS

Regulation of lipoprotein lipase-mediated lipolysis of triglycerides

(2020) Debapriya Basu et al.   CURRENT OPINION IN LIPIDOLOGY

Hyperuricaemia and gout in cardiovascular, metabolic and kidney disease

(2020) Claudio Borghi et al.   European Journal of Internal Medicine

An updated review of pharmacokinetic drug interactions and pharmacogenetics of statins

(2020) Takeshi Hirota et al.   Expert Opinion on Drug Metabolism & Toxicology

Insights into genetic variants associated with NASH-fibrosis from metabolite profiling

(2020) Jake P Mann et al.   HUMAN MOLECULAR GENETICS

Angiopoietin-like protein 3 (ANGPTL3) governs LDL-cholesterol levels through endothelial lipase-dependent VLDL clearance

(2020) Rene C. Adam et al.   JOURNAL OF LIPID RESEARCH

Delivery of Oligonucleotides to the Liver with GalNAc: From Research to Registered Therapeutic Drug

(2020) Alexandre J. Debacker et al.   MOLECULAR THERAPY

Advances in oligonucleotide drug delivery

(2020) Thomas C. Roberts et al.   NATURE REVIEWS DRUG DISCOVERY

Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria

(2020) Manisha Balwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Therapeutic approaches in the treatment of gout

(2020) Michael H. Pillinger et al.   SEMINARS IN ARTHRITIS AND RHEUMATISM

Novel antisense inhibition of diacylglycerol O-acyltransferase 2 for treatment of non-alcoholic fatty liver disease: a multicentre, double-blind, randomised, placebo-controlled phase 2 trial

(2020) Rohit Loomba et al.   Lancet Gastroenterology & Hepatology

LDL-Cholesterol Reduction by ANGPTL3 Inhibition in Mice Is Dependent on Endothelial Lipase

(2020) Liya Wu et al.   CIRCULATION RESEARCH

Vupanorsen, an N-acetyl galactosamine-conjugated antisense drug to ANGPTL3 mRNA, lowers triglycerides and atherogenic lipoproteins in patients with diabetes, hepatic steatosis, and hypertriglyceridaemia

(2020) Daniel Gaudet et al.   EUROPEAN HEART JOURNAL

Genome‐wide association study and meta‐analysis on alcohol‐related liver cirrhosis identifies novel genetic risk factors

(2020) Tae‐Hwi Schwantes‐An et al.   HEPATOLOGY

Evinacumab for Homozygous Familial Hypercholesterolemia

(2020) Frederick J. Raal et al.   NEW ENGLAND JOURNAL OF MEDICINE

Investigating the pharmacodynamic durability of GalNAc–siRNA conjugates

(2020) Christopher R Brown et al.   NUCLEIC ACIDS RESEARCH

Specific ablation of the NCoR corepressor δ splice variant reveals alternative RNA splicing as a key regulator of hepatic metabolism

(2020) Michael L. Goodson et al.   PLoS One

PCSK9 Variants in Familial Hypercholesterolemia: A Comprehensive Synopsis

(2020) Qianyun Guo et al.   Frontiers in Genetics

RNAi targeting liver Angiopoietin-like protein 3 protects from nephrotic syndrome in a rat model via amelioration of pathologic hypertriglyceridemia

(2020) Yitong Zhao et al.   JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS

Evinacumab in Patients with Refractory Hypercholesterolemia

(2020) Robert S. Rosenson et al.   NEW ENGLAND JOURNAL OF MEDICINE

Current and emerging therapies for Duchenne muscular dystrophy and spinal muscular atrophy

(2020) Mohsan Iftikhar et al.   PHARMACOLOGY & THERAPEUTICS

Lipoprotein(a), PCSK9 Inhibition and Cardiovascular Risk: Insights from the FOURIER Trial

(2019) Michelle L. O'Donoghue et al.   CIRCULATION

Gain-of-Function Mutations of SLC16A11 Contribute to the Pathogenesis of Type 2 Diabetes

(2019) Yongxu Zhao et al.   Cell Reports

Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria

(2019) Eliane Sardh et al.   NEW ENGLAND JOURNAL OF MEDICINE

The current state and future directions of RNAi-based therapeutics

(2019) Ryan L. Setten et al.   NATURE REVIEWS DRUG DISCOVERY

Association between PNPLA3 rs738409 polymorphism and nonalcoholic fatty liver disease (NAFLD) susceptibility and severity

(2019) Guangrong Dai et al.   MEDICINE

Selective tissue targeting of synthetic nucleic acid drugs

(2019) Punit P. Seth et al.   JOURNAL OF CLINICAL INVESTIGATION

Effects of Angiopoietin-Like 3 on Triglyceride Regulation, Glucose Homeostasis, and Diabetes

(2019) Eliza Christopoulou et al.   DISEASE MARKERS

Antisense Inhibition of Glucagon Receptor by IONIS-GCGRRx Improves Type 2 Diabetes Without Increase in Hepatic Glycogen Content in Patients With Type 2 Diabetes on Stable Metformin Therapy

(2019) Erin S. Morgan et al.   DIABETES CARE

Genetics of Common, Complex Coronary Artery Disease

(2019) Kiran Musunuru et al.   CELL

Re-Engineering RNA Molecules into Therapeutic Agents

(2019) Martin Egli et al.   ACCOUNTS OF CHEMICAL RESEARCH

Chemical modification of PS-ASO therapeutics reduces cellular protein-binding and improves the therapeutic index

(2019) Wen Shen et al.   NATURE BIOTECHNOLOGY

Accumulation of PNPLA3 on lipid droplets is the basis of associated hepatic steatosis

(2019) Soumik BasuRay et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Pnpla3 silencing with antisense oligonucleotides ameliorates nonalcoholic steatohepatitis and fibrosis in Pnpla3 I148M knock-in mice

(2019) Daniel Lindén et al.   Molecular Metabolism

Glucagon Receptor Signaling and Glucagon Resistance

(2019) Janah et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Inhibition of Angiopoietin-Like Protein 3 With a Monoclonal Antibody Reduces Triglycerides in Hypertriglyceridemia

(2019) Zahid Ahmad et al.   CIRCULATION

2019 ESC/EAS Guidelines for the management of dyslipidaemias: lipid modification to reduce cardiovascular risk

(2019) François Mach et al.   EUROPEAN HEART JOURNAL

Volanesorsen and Triglyceride Levels in Familial Chylomicronemia Syndrome

(2019) Joseph L. Witztum et al.   NEW ENGLAND JOURNAL OF MEDICINE

A NEW DAWN FOR MANAGING DYSLIPIDEMIAS: THE ERA OF RNA-BASED THERAPIES

(2019) C. Macchi et al.   PHARMACOLOGICAL RESEARCH

Clinical review on triglycerides

(2019) Ulrich Laufs et al.   EUROPEAN HEART JOURNAL

The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs

(2019) Akin Akinc et al.   Nature Nanotechnology

A siRNA mediated hepatic dpp4 knockdown affects lipid, but not glucose metabolism in diabetic mice

(2019) Sven Wolfgang Görgens et al.   PLoS One

Ketohexokinase Knockout Mice, a Model for Essential Fructosuria, Exhibit Altered Fructose Metabolism and are Protected from Diet-induced Metabolic Defects

(2018) Corin Miller et al.   AMERICAN JOURNAL OF PHYSIOLOGY-ENDOCRINOLOGY AND METABOLISM

Role of angiopoietin-like 3 (ANGPTL3) in regulating plasma level of low-density lipoprotein cholesterol

(2018) Yu-Xin Xu et al.   ATHEROSCLEROSIS

Is Lp(a) ready for prime time use in the clinic? A pros-and-cons debate

(2018) Karam M. Kostner et al.   ATHEROSCLEROSIS

DGAT2 Inhibition Alters Aspects of Triglyceride Metabolism in Rodents but Not in Non-human Primates

(2018) David G. McLaren et al.   Cell Metabolism

RNA-Targeted Therapeutics

(2018) Stanley T. Crooke et al.   Cell Metabolism

Hydroxysteroid (17β) dehydrogenase 13 deficiency triggers hepatic steatosis and inflammation in mice

(2018) Marion Adam et al.   FASEB JOURNAL

Specific Inhibition of Hepatic Lactate Dehydrogenase Reduces Oxalate Production in Mouse Models of Primary Hyperoxaluria

(2018) Chengjung Lai et al.   MOLECULAR THERAPY

An update on the genetics of hyperuricaemia and gout

(2018) Tanya J. Major et al.   Nature Reviews Rheumatology

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

(2018) David Adams et al.   NEW ENGLAND JOURNAL OF MEDICINE

Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis

(2018) Merrill D. Benson et al.   NEW ENGLAND JOURNAL OF MEDICINE

A Protein-Truncating HSD17B13 Variant and Protection from Chronic Liver Disease

(2018) Noura S. Abul-Husn et al.   NEW ENGLAND JOURNAL OF MEDICINE

Improving siRNA Delivery In Vivo Through Lipid Conjugation

(2018) Maire F. Osborn et al.   Nucleic Acid Therapeutics

Hypertension

(2018) Suzanne Oparil et al.   Nature Reviews Disease Primers

Association of LPA Variants With Risk of Coronary Disease and the Implications for Lipoprotein(a)-Lowering Therapies

(2018) Stephen Burgess et al.   JAMA Cardiology

Lipoprotein(a) and secondary prevention of atherothrombotic events: A critical appraisal

(2018) Michael B. Boffa et al.   Journal of Clinical Lipidology

Effects of APOC3 Heterozygous Deficiency on Plasma Lipid and Lipoprotein Metabolism

(2018) Gissette Reyes-Soffer et al.   ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY

Safety and efficacy of mipomersen in patients with heterozygous familial hypercholesterolemia

(2018) Laurens F. Reeskamp et al.   ATHEROSCLEROSIS

Novel therapeutic approaches in primary hyperoxaluria

(2018) Alexander Weigert et al.   EXPERT OPINION ON EMERGING DRUGS

2018 AHA/ACC/AACVPR/AAPA/ABC/ACPM/ADA/AGS/APhA/ASPC/NLA/PCNA Guideline on the Management of Blood Cholesterol: Executive Summary

(2018) Scott M. Grundy et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Role of HSD17B13 in the liver physiology and pathophysiology

(2018) Wen Su et al.   MOLECULAR AND CELLULAR ENDOCRINOLOGY

Genetics of blood lipids among ~300,000 multi-ethnic participants of the Million Veteran Program

(2018) Derek Klarin et al.   NATURE GENETICS

Alirocumab and Cardiovascular Outcomes after Acute Coronary Syndrome

(2018) Gregory G. Schwartz et al.   NEW ENGLAND JOURNAL OF MEDICINE

Targeted delivery of antisense oligonucleotides to pancreatic β-cells

(2018) C. Ämmälä et al.   Science Advances

Lipoprotein(a): the revenant

(2017) Baris Gencer et al.   EUROPEAN HEART JOURNAL

Acute hepatic porphyrias: Recommendations for evaluation and long-term management

(2017) Manisha Balwani et al.   HEPATOLOGY

Chirality Dependent Potency Enhancement and Structural Impact of Glycol Nucleic Acid Modification on siRNA

(2017) Mark K. Schlegel et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Pathogenic variants that alter protein code often disrupt splicing

(2017) Rachel Soemedi et al.   NATURE GENETICS

Exome-wide association study of plasma lipids in >300,000 individuals

(2017) Dajiang J Liu et al.   NATURE GENETICS

Hypertriglyceridaemia and risk of coronary artery disease

(2017) Željko Reiner   Nature Reviews Cardiology

Angiopoietin-like 3 in lipoprotein metabolism

(2017) Sander Kersten   Nature Reviews Endocrinology

Increasing Lipolysis and Reducing Atherosclerosis

(2017) Alan R. Tall   NEW ENGLAND JOURNAL OF MEDICINE

Evolocumab and Clinical Outcomes in Patients with Cardiovascular Disease

(2017) Marc S. Sabatine et al.   NEW ENGLAND JOURNAL OF MEDICINE

Porphyria

(2017) D. Montgomery Bissell et al.   NEW ENGLAND JOURNAL OF MEDICINE

ANGPTL3 Inhibition in Homozygous Familial Hypercholesterolemia

(2017) Daniel Gaudet et al.   NEW ENGLAND JOURNAL OF MEDICINE

Cardiovascular and Metabolic Effects of ANGPTL3 Antisense Oligonucleotides

(2017) Mark J. Graham et al.   NEW ENGLAND JOURNAL OF MEDICINE

A Highly Durable RNAi Therapeutic Inhibitor of PCSK9

(2017) Kevin Fitzgerald et al.   NEW ENGLAND JOURNAL OF MEDICINE

Genetic and Pharmacologic Inactivation of ANGPTL3 and Cardiovascular Disease

(2017) Frederick E. Dewey et al.   NEW ENGLAND JOURNAL OF MEDICINE

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

(2017) Richard S. Finkel et al.   NEW ENGLAND JOURNAL OF MEDICINE

Impact of enhanced metabolic stability on pharmacokinetics and pharmacodynamics of GalNAc–siRNA conjugates

(2017) Jayaprakash K. Nair et al.   NUCLEIC ACIDS RESEARCH

Myotoxicity of statins: Mechanism of action

(2017) Patrick du Souich et al.   PHARMACOLOGY & THERAPEUTICS

Pharmacokinetic Profiling of Conjugated Therapeutic Oligonucleotides: A High-Throughput Method Based Upon Serial Blood Microsampling Coupled to Peptide Nucleic Acid Hybridization Assay

(2017) Bruno M.D.C. Godinho et al.   Nucleic Acid Therapeutics

Molecular Mechanisms of Antisense Oligonucleotides

(2017) Stanley T. Crooke   Nucleic Acid Therapeutics

Relations between lipoprotein(a) concentrations, LPA genetic variants, and the risk of mortality in patients with established coronary heart disease: a molecular and genetic association study

(2017) Stephen Zewinger et al.   Lancet Diabetes & Endocrinology

Preclinical and Clinical Advances of GalNAc-Decorated Nucleic Acid Therapeutics

(2017) Yuanyu Huang   Molecular Therapy-Nucleic Acids

Synergistic effect of phosphorothioate, 5′-vinylphosphonate and GalNAc modifications for enhancing activity of synthetic siRNA

(2016) Thazha P. Prakash et al.   BIOORGANIC & MEDICINAL CHEMISTRY LETTERS

Update review of the acute porphyrias

(2016) Penelope E. Stein et al.   BRITISH JOURNAL OF HAEMATOLOGY

5′-(E)-Vinylphosphonate: A Stable Phosphate Mimic Can Improve the RNAi Activity of siRNA-GalNAc Conjugates

(2016) Rubina Parmar et al.   CHEMBIOCHEM

Recommendations for Management of Clinically Significant Drug-Drug Interactions With Statins and Select Agents Used in Patients With Cardiovascular Disease: A Scientific Statement From the American Heart Association

(2016) Barbara S. Wiggins et al.   CIRCULATION

Effects of PCSK9 Inhibition With Alirocumab on Lipoprotein Metabolism in Healthy HumansClinical Perspective

(2016) Gissette Reyes-Soffer et al.   CIRCULATION

Global epidemiology of nonalcoholic fatty liver disease-Meta-analytic assessment of prevalence, incidence, and outcomes

(2016) Zobair M. Younossi et al.   HEPATOLOGY

Influence of particle size on the in vivo potency of lipid nanoparticle formulations of siRNA

(2016) Sam Chen et al.   JOURNAL OF CONTROLLED RELEASE

Structure, function, and genetics of lipoprotein (a)

(2016) Konrad Schmidt et al.   JOURNAL OF LIPID RESEARCH

Gout

(2016) Nicola Dalbeth et al.   LANCET

Antisense oligonucleotides targeting apolipoprotein(a) in people with raised lipoprotein(a): two randomised, double-blind, placebo-controlled, dose-ranging trials

(2016) Nicholas J Viney et al.   LANCET

Inhibition of Glycolate Oxidase With Dicer-substrate siRNA Reduces Calcium Oxalate Deposition in a Mouse Model of Primary Hyperoxaluria Type 1

(2016) Chaitali Dutta et al.   MOLECULAR THERAPY

Glycolate Oxidase Is a Safe and Efficient Target for Substrate Reduction Therapy in a Mouse Model of Primary Hyperoxaluria Type I

(2016) Cristina Martin-Higueras et al.   MOLECULAR THERAPY

Small molecules, big targets: drug discovery faces the protein–protein interaction challenge

(2016) Duncan E. Scott et al.   NATURE REVIEWS DRUG DISCOVERY

Hyperoxalurie primitive : une revue de la littérature

(2016) Hassan Bouzidi et al.   Nephrologie & Therapeutique

The delivery of therapeutic oligonucleotides

(2016) Rudolph L. Juliano   NUCLEIC ACIDS RESEARCH

Pharmacokinetic and Pharmacodynamic Investigations of ION-353382, a Model Antisense Oligonucleotide: Using Alpha-2-Macroglobulin and Murinoglobulin Double-Knockout Mice

(2016) Colby S. Shemesh et al.   Nucleic Acid Therapeutics

Dysregulation of RBFOX2 Is an Early Event in Cardiac Pathogenesis of Diabetes

(2016) Curtis A. Nutter et al.   Cell Reports

An alternative splicing program promotes adipose tissue thermogenesis

(2016) Santiago Vernia et al.   eLife

Silencing Myostatin Using Cholesterol-conjugated siRNAs Induces Muscle Growth

(2016) Tayeba Khan et al.   Molecular Therapy-Nucleic Acids

Global Overview of the Epidemiology of Atherosclerotic Cardiovascular Disease

(2015) Simon Barquera et al.   ARCHIVES OF MEDICAL RESEARCH

Thermogenesis in white adipose tissue: An unfinished story about PPARγ

(2015) Guilherme Martins Santos et al.   BIOCHIMICA ET BIOPHYSICA ACTA-GENERAL SUBJECTS

PCSK9 inhibition: the way forward in the treatment of dyslipidemia

(2015) Robert M. Stoekenbroek et al.   BMC Medicine

Hypertension: Renin-Angiotensin-Aldosterone System Alterations

(2015) L. te Riet et al.   CIRCULATION RESEARCH

PCSK9: A key factor modulating atherosclerosis

(2015) Sha Li et al.   Journal of Atherosclerosis and Thrombosis

ANGPTL3 blockade with a human monoclonal antibody reduces plasma lipids in dyslipidemic mice and monkeys

(2015) Viktoria Gusarova et al.   JOURNAL OF LIPID RESEARCH

The Future of Pharmaceutical Manufacturing Sciences

(2015) Jukka Rantanen et al.   JOURNAL OF PHARMACEUTICAL SCIENCES

INS-gene mutations: From genetics and beta cell biology to clinical disease

(2015) Ming Liu et al.   MOLECULAR ASPECTS OF MEDICINE

HIF-driven SF3B1 induces KHK-C to enforce fructolysis and heart disease

(2015) Peter Mirtschink et al.   NATURE

LTB4 promotes insulin resistance in obese mice by acting on macrophages, hepatocytes and myocytes

(2015) Pingping Li et al.   NATURE MEDICINE

Management of patients with familial hypercholesterolaemia

(2015) Željko Reiner   Nature Reviews Cardiology

Hyperinsulinemic Hypoglycemia

(2015) Maria Güemes et al.   PEDIATRIC CLINICS OF NORTH AMERICA

Acute Porphyrias in the USA: Features of 108 Subjects from Porphyrias Consortium

(2014) Herbert L. Bonkovsky et al.   AMERICAN JOURNAL OF MEDICINE

The Global Burden of Ischemic Heart Disease in 1990 and 2010

(2014) Andrew E. Moran et al.   CIRCULATION

Glucagon and Type 2 Diabetes: the Return of the Alpha Cell

(2014) Asger Lund et al.   Current Diabetes Reports

Homozygous familial hypercholesterolaemia: new insights and guidance for clinicians to improve detection and clinical management. A position paper from the Consensus Panel on Familial Hypercholesterolaemia of the European Atherosclerosis Society

(2014) M. Cuchel et al.   EUROPEAN HEART JOURNAL

Liver transplantation in the management of porphyria

(2014) Ashwani K. Singal et al.   HEPATOLOGY

The mammalian molybdenum enzymes of mARC

(2014) Gudrun Ott et al.   JOURNAL OF BIOLOGICAL INORGANIC CHEMISTRY

Negative Regulators of Brown Adipose Tissue (BAT)-Mediated Thermogenesis

(2014) Bal Krishan Sharma et al.   JOURNAL OF CELLULAR PHYSIOLOGY

Mutations inHAO1encoding glycolate oxidase cause isolated glycolic aciduria

(2014) Yaacov Frishberg et al.   JOURNAL OF MEDICAL GENETICS

Multivalent N-Acetylgalactosamine-Conjugated siRNA Localizes in Hepatocytes and Elicits Robust RNAi-Mediated Gene Silencing

(2014) Jayaprakash K. Nair et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Reduction in Lipoprotein(a) With PCSK9 Monoclonal Antibody Evolocumab (AMG 145)

(2014) Frederick J. Raal et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Alteration of NCoR Corepressor Splicing in Mice Causes Increased Body Weight and Hepatosteatosis without Glucose Intolerance

(2014) M. L. Goodson et al.   MOLECULAR AND CELLULAR BIOLOGY

Loss-of-Function Mutations in APOC3 and Risk of Ischemic Vascular Disease

(2014) Anders Berg Jørgensen et al.   NEW ENGLAND JOURNAL OF MEDICINE

Loss-of-Function Mutations in APOC3, Triglycerides, and Coronary Disease

(2014)   NEW ENGLAND JOURNAL OF MEDICINE

Targeted delivery of antisense oligonucleotides to hepatocytes using triantennaryN-acetyl galactosamine improves potency 10-fold in mice

(2014) Thazha P. Prakash et al.   NUCLEIC ACIDS RESEARCH

Comparative proteomic study reveals 17 -HSD13 as a pathogenic protein in nonalcoholic fatty liver disease

(2014) W. Su et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

RNAi-mediated silencing of hepatic Alas1 effectively prevents and treats the induced acute attacks in acute intermittent porphyria mice

(2014) M. Yasuda et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

The role of glucagon on type 2 diabetes at a glance

(2014) Amélio F Godoy-Matos   Diabetology & Metabolic Syndrome

Phosphorothioates, Essential Components of Therapeutic Oligonucleotides

(2014) Fritz Eckstein   Nucleic Acid Therapeutics

Discontinuation of Statins in Routine Care Settings

(2013) Huabing Zhang et al.   ANNALS OF INTERNAL MEDICINE

Drug reaction with eosinophilia and systemic symptoms (DRESS): an original multisystem adverse drug reaction. Results from the prospective RegiSCAR study

(2013) S.H. Kardaun et al.   BRITISH JOURNAL OF DERMATOLOGY

Antisense Oligonucleotide Inhibition of Apolipoprotein C-III Reduces Plasma Triglycerides in Rodents, Nonhuman Primates, and Humans

(2013) Mark J. Graham et al.   CIRCULATION RESEARCH

Targeting of hepatic angiotensinogen using chemically modified siRNAs results in significant and sustained blood pressure lowering in a rat model of hypertension

(2013) Jeffrey Olearczyk et al.   HYPERTENSION RESEARCH

Integrated guidance on the care of familial hypercholesterolaemia from the International FH Foundation

(2013) Gerald F. Watts et al.   INTERNATIONAL JOURNAL OF CARDIOLOGY

Reactivation of Fetal Splicing Programs in Diabetic Hearts Is Mediated by Protein Kinase C Signaling

(2013) Sunil K. Verma et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Elevated Lipoprotein(a) and Risk of Aortic Valve Stenosis in the General Population

(2013) Pia R. Kamstrup et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Biodegradable Lipids Enabling Rapidly Eliminated Lipid Nanoparticles for Systemic Delivery of RNAi Therapeutics

(2013) Martin A Maier et al.   MOLECULAR THERAPY

Sequence variants in SLC16A11 are a common risk factor for type 2 diabetes in Mexico

(2013)   NATURE

Molecular dissection of human Argonaute proteins by DNA shuffling

(2013) Nina Schürmann et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

The porphyrias: advances in diagnosis and treatment

(2012) M. Balwani et al.   BLOOD

Characteristics and Outcomes of Children with Primary Oxalosis Requiring Renal Replacement Therapy

(2012) J. Harambat et al.   Clinical Journal of the American Society of Nephrology

NMR study of ligand release from asialoglycoprotein receptor under solution conditions in early endosomes

(2012) Takuo Onizuka et al.   FEBS Journal

Guidelines for the Management of Familial Hypercholesterolemia

(2012) Mariko Harada-Shiba et al.   Journal of Atherosclerosis and Thrombosis

Furin-cleaved Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) Is Active and Modulates Low Density Lipoprotein Receptor and Serum Cholesterol Levels

(2012) Michael T. Lipari et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

RNA therapeutics: beyond RNA interference and antisense oligonucleotides

(2012) Ryszard Kole et al.   NATURE REVIEWS DRUG DISCOVERY

An update on primary hyperoxaluria

(2012) Bernd Hoppe   Nature Reviews Nephrology

Primary hyperoxaluria Type 1: indications for screening and guidance for diagnosis and treatment

(2012) P. Cochat et al.   NEPHROLOGY DIALYSIS TRANSPLANTATION

Randomized, Placebo-Controlled Trial of Mipomersen in Patients with Severe Hypercholesterolemia Receiving Maximally Tolerated Lipid-Lowering Therapy

(2012) Mary P. McGowan et al.   PLoS One

Manipulation of PK-M mutually exclusive alternative splicing by antisense oligonucleotides

(2012) Z. Wang et al.   Open Biology

Ethnic Differences in Hypertension Incidence Among Middle-Aged and Older Adults

(2011) April P. Carson et al.   HYPERTENSION

Alternative mRNA Splicing of Corepressors Generates Variants That Play Opposing Roles in Adipocyte Differentiation

(2011) Michael L. Goodson et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Biodistribution of Small Interfering RNA at the Organ and Cellular Levels after Lipid Nanoparticle-mediated Delivery

(2011) Bin Shi et al.   JOURNAL OF HISTOCHEMISTRY & CYTOCHEMISTRY

Using positional distribution to identify splicing elements and predict pre-mRNA processing defects in human genes

(2011) K. H. Lim et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Exon-centric regulation of pyruvate kinase M alternative splicing via mutually exclusive exons

(2011) Zhenxun Wang et al.   Journal of Molecular Cell Biology

Angiopoietin-like Protein 3 Inhibits Lipoprotein Lipase Activity through Enhancing Its Cleavage by Proprotein Convertases

(2010) Jun Liu et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

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

(2010) Frederick J Raal et al.   LANCET

Targeted Delivery of RNAi Therapeutics With Endogenous and Exogenous Ligand-Based Mechanisms

(2010) Akin Akinc et al.   MOLECULAR THERAPY

Rational design of cationic lipids for siRNA delivery

(2010) Sean C Semple et al.   NATURE BIOTECHNOLOGY

Exome Sequencing,ANGPTL3Mutations, and Familial Combined Hypolipidemia

(2010) Kiran Musunuru et al.   NEW ENGLAND JOURNAL OF MEDICINE

The alternative splicing repressors hnRNP A1/A2 and PTB influence pyruvate kinase isoform expression and cell metabolism

(2010) C. V. Clower et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Genetically Elevated Lipoprotein(a) and Increased Risk of Myocardial Infarction

(2009) Pia R. Kamstrup   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Consuming fructose-sweetened, not glucose-sweetened, beverages increases visceral adiposity and lipids and decreases insulin sensitivity in overweight/obese humans

(2009) Kimber L. Stanhope et al.   JOURNAL OF CLINICAL INVESTIGATION

Genetic Variants Associated with Lp(a) Lipoprotein Level and Coronary Disease

(2009) Robert Clarke et al.   NEW ENGLAND JOURNAL OF MEDICINE

A large-scale chemical modification screen identifies design rules to generate siRNAs with high activity, high stability and low toxicity

(2009) Jesper B. Bramsen et al.   NUCLEIC ACIDS RESEARCH

A proprotein convertase subtilisin/kexin type 9 neutralizing antibody reduces serum cholesterol in mice and nonhuman primates

(2009) J. C. Y. Chan et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

The Asialoglycoprotein Receptor Regulates Levels of Plasma Glycoproteins Terminating with Sialic Acid α2,6-Galactose

(2008) Lindsay M. Steirer et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

PCSK9: a convertase that coordinates LDL catabolism

(2008) Jay D. Horton et al.   JOURNAL OF LIPID RESEARCH

Global burden of blood-pressure-related disease, 2001

(2008) Carlene MM Lawes et al.   LANCET

Sequence- and target-independent angiogenesis suppression by siRNA via TLR3

(2008) Mark E. Kleinman et al.   NATURE

Genetic variation in PNPLA3 confers susceptibility to nonalcoholic fatty liver disease

(2008) Stefano Romeo et al.   NATURE GENETICS

Newly identified loci that influence lipid concentrations and risk of coronary artery disease

(2008) Cristen J Willer et al.   NATURE GENETICS

10-Year Follow-up of Intensive Glucose Control in Type 2 Diabetes

(2008) Rury R. Holman et al.   NEW ENGLAND JOURNAL OF MEDICINE

Therapeutic RNAi targeting PCSK9 acutely lowers plasma cholesterol in rodents and LDL cholesterol in nonhuman primates

(2008) M. Frank-Kamenetsky et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A Null Mutation in Human APOC3 Confers a Favorable Plasma Lipid Profile and Apparent Cardioprotection

(2008) T. I. Pollin et al.   SCIENCE

Allopurinol is the most common cause of Stevens-Johnson syndrome and toxic epidermal necrolysis in Europe and Israel

(2007) Sima Halevy et al.   JOURNAL OF THE AMERICAN ACADEMY OF DERMATOLOGY