Gene Therapy Targeting PCSK9

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

PCSK9: a view beyond the canonical cholesterol lowering impact

(2021) Chiara Macchi et al.   AMERICAN JOURNAL OF PATHOLOGY

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

Prime editing – an update on the field

(2021) Janine Scholefield et al.   GENE THERAPY

Global perspective of familial hypercholesterolaemia: a cross-sectional study from the EAS Familial Hypercholesterolaemia Studies Collaboration (FHSC)

(2021) Antonio J. Vallejo-Vaz et al.   LANCET

The Multifaceted Biology of PCSK9

(2021) Nabil G Seidah et al.   ENDOCRINE REVIEWS

Inclisiran: a small interfering RNA strategy targeting PCSK9 to treat hypercholesterolemia

(2021) Yajnavalka Banerjee et al.   Expert Opinion On Drug Safety

CRISPR Gene Editing in Lipid Disorders and Atherosclerosis: Mechanisms and Opportunities

(2021) Harry E. Walker et al.   Metabolites

Prevalence of Familial Hypercholesterolemia Among the General Population and Patients With Atherosclerotic Cardiovascular Disease

(2020) Pengwei Hu et al.   CIRCULATION

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

Targeting RNA With Antisense Oligonucleotides and Small Interfering RNA in Dyslipidemias

(2020) Julius L. Katzmann et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Latest Developed Strategies to Minimize the Off-Target Effects in CRISPR-Cas-Mediated Genome Editing

(2020) Muhammad Naeem et al.   Cells

CRISPR-Cas9 DNA Base-Editing and Prime-Editing

(2020) Ariel Kantor et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Base editing: advances and therapeutic opportunities

(2020) Elizabeth M. Porto et al.   NATURE REVIEWS DRUG DISCOVERY

Various Aspects of a Gene Editing System—CRISPR–Cas9

(2020) Edyta Janik et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

PCSK9 Inhibition: Insights From Clinical Trials and Future Prospects

(2020) Julius L. Katzmann et al.   Frontiers in Physiology

Novel strategies to target PCSK9: beyond monoclonal antibodies

(2019) Nabil G Seidah et al.   CARDIOVASCULAR RESEARCH

Association of Statin Adherence With Mortality in Patients With Atherosclerotic Cardiovascular Disease

(2019) Fatima Rodriguez et al.   JAMA Cardiology

A review of gene- and cell-based therapies for familial hypercholesterolemia

(2019) Saeideh Hajighasemi et al.   PHARMACOLOGICAL RESEARCH

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

(2019) François Mach et al.   EUROPEAN HEART JOURNAL

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

(2019) C. Macchi et al.   PHARMACOLOGICAL RESEARCH

Reduced Blood Lipid Levels With In Vivo CRISPR-Cas9 Base Editing of ANGPTL3

(2018) Alexandra C. Chadwick et al.   CIRCULATION

Delivering CRISPR: a review of the challenges and approaches

(2018) Christopher A. Lino et al.   DRUG DELIVERY

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

(2018) Lili Wang et al.   NATURE BIOTECHNOLOGY

Alirocumab and Cardiovascular Outcomes after Acute Coronary Syndrome

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

Vaccination to prevent atherosclerotic cardiovascular diseases

(2017) Ulrich Laufs et al.   EUROPEAN HEART JOURNAL

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

Evolocumab and Clinical Outcomes in Patients with Cardiovascular Disease

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

CRISPR-Cas9 Genome Editing for Treatment of Atherogenic Dyslipidemia

(2017) Alexandra C. Chadwick et al.   ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY

CRISPR/Cas9 in Genome Editing and Beyond

(2016) Haifeng Wang et al.   Annual Review of Biochemistry

CRISPR-Cas9 Targeting ofPCSK9in Human Hepatocytes In Vivo—Brief ReportSignificance

(2016) Xiao Wang et al.   ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY

Genome editing in cardiovascular diseases

(2016) Alanna Strong et al.   Nature Reviews Cardiology

CRISPR-Cas9: a new and promising player in gene therapy

(2015) Lu Xiao-Jie et al.   JOURNAL OF MEDICAL GENETICS

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

Permanent Alteration of PCSK9 With In Vivo CRISPR-Cas9 Genome Editing

(2014) Qiurong Ding et al.   CIRCULATION RESEARCH

CRISPR/Cas9 for genome editing: progress, implications and challenges

(2014) F. Zhang et al.   HUMAN MOLECULAR GENETICS

PCSK9 Inhibition - A Novel Mechanism to Treat Lipid Disorders?

(2013) Maciej Banach et al.   CURRENT PHARMACEUTICAL DESIGN

RNA-guided genetic silencing systems in bacteria and archaea

(2012) Blake Wiedenheft et al.   NATURE

Cas9-crRNA ribonucleoprotein complex mediates specific DNA cleavage for adaptive immunity in bacteria

(2012) G. Gasiunas et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

(2012) M. Jinek et al.   SCIENCE

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

(2010) Akin Akinc et al.   MOLECULAR THERAPY