In vivo genome and base editing of a human PCSK9 knock-in hypercholesterolemic mouse model

Improving cytidine and adenine base editors by expression optimization and ancestral reconstruction

(2018) Luke W Koblan et al.   NATURE BIOTECHNOLOGY

Optimized base editors enable efficient editing in cells, organoids and mice

(2018) Maria Paz Zafra et al.   NATURE BIOTECHNOLOGY

In vivo CRISPR editing with no detectable genome-wide off-target mutations

(2018) Pinar Akcakaya et al.   NATURE

In utero CRISPR-mediated therapeutic editing of metabolic genes

(2018) Avery C. Rossidis et al.   NATURE MEDICINE

Successful treatment of a patient with statin-induced myopathy and myotonic dystrophy type II with proprotein convertase subtilisin/kexin type 9 inhibitor, alirocumab (Praluent)

(2017) Mohamed K.M. Shakir et al.   Journal of Clinical Lipidology

Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage

(2017) Nicole M. Gaudelli et al.   NATURE

Highly efficient RNA-guided base editing in mouse embryos

(2017) Kyoungmi Kim et al.   NATURE BIOTECHNOLOGY

Genome-wide target specificities of CRISPR RNA-guided programmable deaminases

(2017) Daesik Kim et al.   NATURE BIOTECHNOLOGY

A Highly Durable RNAi Therapeutic Inhibitor of PCSK9

(2017)   NEW ENGLAND JOURNAL OF MEDICINE

Improved base excision repair inhibition and bacteriophage Mu Gam protein yields C:G-to-T:A base editors with higher efficiency and product purity

(2017) Alexis C. Komor et al.   Science Advances

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

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

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

(2016) Alexis C. Komor et al.   NATURE

Targeted nucleotide editing using hybrid prokaryotic and vertebrate adaptive immune systems

(2016) K. Nishida et al.   SCIENCE

Effects of Proprotein Convertase Subtilisin/Kexin Type 9 Antibodies in Adults With Hypercholesterolemia

(2015) Eliano Pio Navarese et al.   ANNALS OF INTERNAL MEDICINE

Characterization of Autosomal Dominant Hypercholesterolemia Caused byPCSK9Gain of Function Mutations and Its Specific Treatment With Alirocumab, a PCSK9 Monoclonal AntibodyCLINICAL PERSPECTIVE

(2015) Paul N. Hopkins et al.   Circulation-Cardiovascular Genetics

Efficacy and safety of alirocumab vs ezetimibe in statin-intolerant patients, with a statin rechallenge arm: The ODYSSEY ALTERNATIVE randomized trial

(2015) Patrick M. Moriarty et al.   Journal of Clinical Lipidology

Characterization of the First PCSK9 Gain of Function Homozygote

(2015) Ana Catarina Alves et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Inhibition of PCSK9 with evolocumab in homozygous familial hypercholesterolaemia (TESLA Part B): a randomised, double-blind, placebo-controlled trial

(2015) Frederick J Raal et al.   LANCET

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

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

Characterization of Autosomal Dominant Hypercholesterolemia Caused byPCSK9Gain of Function Mutations and Its Specific Treatment With Alirocumab, a PCSK9 Monoclonal AntibodyCLINICAL PERSPECTIVE

(2015) Paul N. Hopkins et al.   Circulation-Cardiovascular Genetics

Statin Intolerance: Diagnosis and Remedies

(2015) Angela Pirillo et al.   Current Cardiology Reports

Genotypic and phenotypic features in homozygous familial hypercholesterolemia caused by proprotein convertase subtilisin/kexin type 9 ( PCSK9 ) gain-of-function mutation

(2014) Hiroshi Mabuchi et al.   ATHEROSCLEROSIS

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

(2014) Qiurong Ding et al.   CIRCULATION RESEARCH

Efficacy and safety of alirocumab, a monoclonal antibody to PCSK9, in statin-intolerant patients: Design and rationale of ODYSSEY ALTERNATIVE, a randomized phase 3 trial

(2014) Patrick M. Moriarty et al.   Journal of Clinical Lipidology

PCSK9 inhibition fails to alter hepatic LDLR, circulating cholesterol, and atherosclerosis in the absence of ApoE

(2014) Brandon Ason et al.   JOURNAL OF LIPID RESEARCH

Anti-PCSK9 Antibody Effectively Lowers Cholesterol in Patients With Statin Intolerance

(2014) Erik Stroes et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Chromosomal Translocations in Human Cells Are Generated by Canonical Nonhomologous End-Joining

(2014) Hind Ghezraoui et al.   MOLECULAR CELL

GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases

(2014) Shengdar Q Tsai et al.   NATURE BIOTECHNOLOGY

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

Identification and characterization of new gain-of-function mutations in the PCSK9 gene responsible for autosomal dominant hypercholesterolemia

(2012) Marianne Abifadel et al.   ATHEROSCLEROSIS

Association of LDL Cholesterol, Non–HDL Cholesterol, and Apolipoprotein B Levels With Risk of Cardiovascular Events Among Patients Treated With Statins

(2012) S. Matthijs Boekholdt et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

A Meta-Analysis of Low-Density Lipoprotein Cholesterol, Non-High-Density Lipoprotein Cholesterol, and Apolipoprotein B as Markers of Cardiovascular Risk

(2011) Allan D. Sniderman et al.  

PCSK9: an emerging target for treatment of hypercholesterolemia

(2011) Christopher J Duff et al.   EXPERT OPINION ON THERAPEUTIC TARGETS

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

Analyzing of high-density lipoprotein subfractions and low-density lipoprotein subfractions in human serum with anion-exchange chromatography

(2008) Yuji Hirowatari et al.   ATHEROSCLEROSIS