New Approaches for Targeting PCSK9: Small-Interfering Ribonucleic Acid and Genome Editing

CRISPR technology: A decade of genome editing is only the beginning

(2023) Joy Y. Wang et al.   SCIENCE

Association of Rare Protein-Truncating DNA Variants in APOB or PCSK9 With Low-density Lipoprotein Cholesterol Level and Risk of Coronary Heart Disease

(2023) Jacqueline S. Dron et al.   JAMA Cardiology

Drug delivery systems for RNA therapeutics

(2022) Kalina Paunovska et al.   NATURE REVIEWS GENETICS

Efficacy and Safety of an Investigational Single-course CRISPR Base Editing Therapy Targeting PCSK9 in Non-human Primate and Mouse Models

(2022) Richard G. Lee et al.   CIRCULATION

An in vivo CRISPR base editing therapy to inactivate the ANGPTL3 gene: nomination of a development candidate for VERVE-201

(2022) A Khera et al.   EUROPEAN HEART JOURNAL

2022 ACC Expert Consensus Decision Pathway on the Role of Nonstatin Therapies for LDL-Cholesterol Lowering in the Management of Atherosclerotic Cardiovascular Disease Risk

(2022) Donald M. Lloyd-Jones et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Effect of statin therapy on muscle symptoms: an individual participant data meta-analysis of large-scale, randomised, double-blind trials

(2022) Christina Reith et al.   LANCET

LDL-C target attainment in secondary prevention of ASCVD in the United States: barriers, consequences of nonachievement, and strategies to reach goals

(2022) James Underberg et al.   POSTGRADUATE MEDICINE

RNA therapeutics: updates and future potential

(2022) Caroline Zhang et al.   Science China-Life Sciences

Long-term stable reduction of low-density lipoprotein in nonhuman primates following in vivo genome editing of PCSK9

(2021) Lili Wang et al.   MOLECULAR THERAPY

Sequencing of 53,831 diverse genomes from the NHLBI TOPMed Program

(2021) Daniel Taliun et al.   NATURE

In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates

(2021) Kiran Musunuru et al.   NATURE

In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels

(2021) Tanja Rothgangl et al.   NATURE BIOTECHNOLOGY

Adherence and persistence in the use of statins and ezetimibe over 8 years in a real-life study

(2021) Fiorenzo Santoleri et al.   CURRENT MEDICAL RESEARCH AND OPINION

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

(2021) Julian D. Gillmore et al.   NEW ENGLAND JOURNAL OF MEDICINE

Pre-Clinical Evaluation of the Nanoliposomal antiPCSK9 Vaccine in Healthy Non-Human Primates

(2021) Amir Abbas Momtazi-Borojeni et al.   Vaccines

ACC/AHA lipid guidelines: Personalized care to prevent cardiovascular disease

(2020) Cara Reiter-Brennan et al.   CLEVELAND CLINIC JOURNAL OF MEDICINE

The mutational constraint spectrum quantified from variation in 141,456 humans

(2020) Konrad J. Karczewski et al.   NATURE

Directed evolution of adenine base editors with increased activity and therapeutic application

(2020) Nicole M. Gaudelli et al.   NATURE BIOTECHNOLOGY

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

Genome editing with CRISPR–Cas nucleases, base editors, transposases and prime editors

(2020) Andrew V. Anzalone et al.   NATURE BIOTECHNOLOGY

Efficacy and Safety of PCSK9 Inhibition With Evolocumab in Reducing Cardiovascular Events in Patients With Metabolic Syndrome Receiving Statin Therapy

(2020) Prakash Deedwania et al.   JAMA Cardiology

Effect of alirocumab on major adverse cardiovascular events according to renal function in patients with a recent acute coronary syndrome: prespecified analysis from the ODYSSEY OUTCOMES randomized clinical trial

(2020) José Tuñón et al.   EUROPEAN HEART JOURNAL

Time Course of LDL Cholesterol Exposure and Cardiovascular Disease Event Risk

(2020) Michael J. Domanski et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

CRISPR/Cas9: A preclinical and clinical perspective for the treatment of human diseases

(2020) Garima Sharma et al.   MOLECULAR THERAPY

siRNA based drug design, quality, delivery and clinical translation

(2020) Md Abdus Subhan et al.   Nanomedicine-Nanotechnology Biology and Medicine

CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

(2020) Haydar Frangoul et al.   NEW ENGLAND JOURNAL OF MEDICINE

Association of Genetic Variants Related to Combined Exposure to Lower Low-Density Lipoproteins and Lower Systolic Blood Pressure With Lifetime Risk of Cardiovascular Disease

(2019) Brian A. Ference et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Therapeutic effect of nanoliposomal PCSK9 vaccine in a mouse model of atherosclerosis

(2019) Amir Abbas Momtazi-Borojeni et al.   BMC Medicine

Prior Authorization Requirements for Proprotein Convertase Subtilisin/Kexin Type 9 Inhibitors Across US Private and Public Payers

(2018) Jalpa A. Doshi et al.  

PCSK9 deficiency reduces insulin secretion and promotes glucose intolerance: the role of the low-density lipoprotein receptor

(2018) Lorenzo Da Dalt et al.   EUROPEAN HEART JOURNAL

Meganuclease targeting of PCSK9 in macaque liver leads to stable reduction in serum cholesterol

(2018) Lili Wang et al.   NATURE BIOTECHNOLOGY

Prior Authorization Requirements for Proprotein Convertase Subtilisin/Kexin Type 9 Inhibitors Across US Private and Public Payers

(2018) Jalpa A. Doshi et al.   Circulation-Cardiovascular Quality and Outcomes

Alirocumab and Cardiovascular Outcomes after Acute Coronary Syndrome

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

Low-density lipoproteins cause atherosclerotic cardiovascular disease. 1. Evidence from genetic, epidemiologic, and clinical studies. A consensus statement from the European Atherosclerosis Society Consensus Panel

(2017) Brian A. Ference et al.   EUROPEAN HEART JOURNAL

The AT04A vaccine against proprotein convertase subtilisin/kexin type 9 reduces total cholesterol, vascular inflammation, and atherosclerosis in APOE*3Leiden.CETP mice

(2017) Christine Landlinger et al.   EUROPEAN HEART JOURNAL

Evolocumab and Clinical Outcomes in Patients with Cardiovascular Disease

(2017) Marc S. Sabatine 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

Preclinical evaluation of RNAi as a treatment for transthyretin-mediated amyloidosis

(2016) James S. Butler et al.   AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS

Reduction in Total Cardiovascular Events With Ezetimibe/Simvastatin Post-Acute Coronary Syndrome

(2016) Sabina A. Murphy et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage

(2016) Alexis C. Komor et al.   NATURE

Fibronectin-based scaffold domain proteins that bind myostatin: a patent evaluation of WO2014043344

(2015) Ryan G Walker et al.   EXPERT OPINION ON THERAPEUTIC PATENTS

Efficacy and Safety of Alirocumab in Reducing Lipids and Cardiovascular Events

(2015) Jennifer G. Robinson et al.   NEW ENGLAND JOURNAL OF MEDICINE

Efficacy and Safety of Evolocumab in Reducing Lipids and Cardiovascular Events

(2015) Marc S. Sabatine et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

Very Low Levels of Atherogenic Lipoproteins and the Risk for Cardiovascular Events

(2014) S. Matthijs Boekholdt et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

LDL CHOLESTEROL REDUCTION WITH BMS-962476, AN ADNECTIN INHIBITOR OF PCSK9: RESULTS OF A SINGLE ASCENDING DOSE STUDY

(2014) Evan A. Stein et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

The effects of lowering LDL cholesterol with statin therapy in people at low risk of vascular disease: meta-analysis of individual data from 27 randomised trials

(2013)   LANCET

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

(2013) Kevin Fitzgerald et al.   LANCET

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

(2012) M. Jinek et al.   SCIENCE

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