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

Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing

(2017) Hao Yin et al.   NATURE BIOTECHNOLOGY

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

(2017) Jerry R. Mendell et al.   NEW ENGLAND JOURNAL OF MEDICINE

AAV5–Factor VIII Gene Transfer in Severe Hemophilia A

(2017) Savita Rangarajan et al.   NEW ENGLAND JOURNAL OF MEDICINE

Class I-restricted T-cell responses to a polymorphic peptide in a gene therapy clinical trial for α-1-antitrypsin deficiency

(2017) Roberto Calcedo et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Non-Clinical Study Examining AAV8.TBG.hLDLR Vector-Associated Toxicity in Chow-Fed Wild-Type and LDLR+/− Rhesus Macaques

(2017) Jenny A. Greig et al.   Human Gene Therapy Clinical Development

Je, a versatile suite to handle multiplexed NGS libraries with unique molecular identifiers

(2016) Charles Girardot et al.   BMC BIOINFORMATICS

Open-source guideseq software for analysis of GUIDE-seq data

(2016) Shengdar Q Tsai et al.   NATURE BIOTECHNOLOGY

Genome-wide specificities of CRISPR-Cas Cpf1 nucleases in human cells

(2016) Benjamin P Kleinstiver et al.   NATURE BIOTECHNOLOGY

A multifunctional AAV–CRISPR–Cas9 and its host response

(2016) Wei Leong Chew et al.   NATURE METHODS

The NAB2–STAT6 gene fusion in solitary fibrous tumor can be reliably detected by anchored multiplexed PCR for targeted next-generation sequencing

(2016) Natalya V. Guseva et al.   Cancer Genetics

Novel CAD-ALK gene rearrangement is drugable by entrectinib in colorectal cancer

(2015) Alessio Amatu et al.   BRITISH JOURNAL OF CANCER

PCSK9 inhibition with evolocumab (AMG 145) in heterozygous familial hypercholesterolaemia (RUTHERFORD-2): a randomised, double-blind, placebo-controlled trial

(2015) Frederick J Raal et al.   LANCET

Transient Expression of Virally Delivered Meganuclease In Planta Generates Inherited Genomic Deletions

(2015) Arik Honig et al.   Molecular Plant

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy

(2015) C. Long et al.   SCIENCE

In vivo gene editing in dystrophic mouse muscle and muscle stem cells

(2015) M. Tabebordbar et al.   SCIENCE

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

(2015) C. E. Nelson et al.   SCIENCE

Role of MTA2 in human cancer

(2014) Kyle R. Covington et al.   CANCER AND METASTASIS REVIEWS

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

(2014) Qiurong Ding et al.   CIRCULATION RESEARCH

Lentiviral protein delivery of meganucleases in human cells mediates gene targeting and alleviates toxicity

(2014) C He et al.   GENE THERAPY

Promoterless gene targeting without nucleases ameliorates haemophilia B in mice

(2014) A. Barzel et al.   NATURE

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

(2014) Shengdar Q Tsai et al.   NATURE BIOTECHNOLOGY

Anchored multiplex PCR for targeted next-generation sequencing

(2014) Zongli Zheng et al.   NATURE MEDICINE

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

(2014) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

PEAR: a fast and accurate Illumina Paired-End reAd mergeR

(2013) J. Zhang et al.   BIOINFORMATICS

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

Absolute Determination of Single-Stranded and Self-Complementary Adeno-Associated Viral Vector Genome Titers by Droplet Digital PCR

(2013) Martin Lock et al.   Human Gene Therapy Methods

Targeted DNA excision in Arabidopsis by a re-engineered homing endonuclease

(2012) Mauricio S Antunes et al.   BMC BIOTECHNOLOGY

Self-Renewing Endodermal Progenitor Lines Generated from Human Pluripotent Stem Cells

(2012) Xin Cheng et al.   Cell Stem Cell

Generation of Rag1-knockout immunodeficient rats and mice using engineered meganucleases

(2012) Séverine Ménoret et al.   FASEB JOURNAL

Inhibition of PCSK9 in familial hypercholesterolaemia

(2012) Eric JG Sijbrands   LANCET

Fast, scalable generation of high-quality protein multiple sequence alignments using Clustal Omega

(2011) F. Sievers et al.   Molecular Systems Biology

Systemic Administration of AAV8-α-galactosidase A Induces Humoral Tolerance in Nonhuman Primates Despite Low Hepatic Expression

(2011) Jennifer B Nietupski et al.   MOLECULAR THERAPY

In vivo genome editing restores haemostasis in a mouse model of haemophilia

(2011) Hojun Li et al.   NATURE

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

(2011) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Rapid, Simple, and Versatile Manufacturing of Recombinant Adeno-Associated Viral Vectors at Scale

(2010) Martin Lock et al.   HUMAN GENE THERAPY

Generation of Transgene-Free Lung Disease-Specific Human Induced Pluripotent Stem Cells Using a Single Excisable Lentiviral Stem Cell Cassette

(2010) Aba Somers et al.   STEM CELLS

The Sequence Alignment/Map format and SAMtools

(2009) H. Li et al.   BIOINFORMATICS

Fast and accurate short read alignment with Burrows-Wheeler transform

(2009) H. Li et al.   BIOINFORMATICS

Adeno-Associated Virus-Mediated Gene Transfer to Nonhuman Primate Liver Can Elicit Destructive Transgene-Specific T Cell Responses

(2009) Guangping Gao et al.   HUMAN GENE THERAPY

Genetic and Metabolic Determinants of Plasma PCSK9 Levels

(2009) Susan G. Lakoski et al.   JOURNAL OF CLINICAL ENDOCRINOLOGY & METABOLISM

Worldwide Epidemiology of Neutralizing Antibodies to Adeno‐Associated Viruses

(2009) Roberto Calcedo et al.   JOURNAL OF INFECTIOUS DISEASES

Generation of redesigned homing endonucleases comprising DNA-binding domains derived from two different scaffolds

(2009) Sylvestre Grizot et al.   NUCLEIC ACIDS RESEARCH

Efficient targeting of a SCID gene by an engineered single-chain homing endonuclease

(2009) Sylvestre Grizot et al.   NUCLEIC ACIDS RESEARCH

PCSK9: a convertase that coordinates LDL catabolism

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

Host Immune Responses to Chronic Adenovirus Infections in Human and Nonhuman Primates

(2008) R. Calcedo et al.   JOURNAL OF VIROLOGY

Molecular basis of xeroderma pigmentosum group C DNA recognition by engineered meganucleases

(2008) Pilar Redondo et al.   NATURE