Application of prime editing to the correction of mutations and phenotypes in adult mice with liver and eye diseases

Engineered prime editors with PAM flexibility

(2021) Jiyeon Kweon et al.   MOLECULAR THERAPY

CRISPR prime editing with ribonucleoprotein complexes in zebrafish and primary human cells

(2021) Karl Petri et al.   NATURE BIOTECHNOLOGY

Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice

(2021) Pengpeng Liu et al.   Nature Communications

Engineering adeno-associated virus vectors for gene therapy

(2020) Chengwen Li et al.   NATURE REVIEWS GENETICS

High-throughput analysis of the activities of xCas9, SpCas9-NG and SpCas9 at matched and mismatched target sequences in human cells

(2020) Hui Kwon Kim et al.   Nature Biomedical Engineering

Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses

(2020) Jonathan M. Levy et al.   Nature Biomedical Engineering

Prime genome editing in rice and wheat

(2020) Qiupeng Lin et al.   NATURE BIOTECHNOLOGY

Efficient generation of mouse models with the prime editing system

(2020) Yao Liu et al.   Cell Discovery

Sequence-specific prediction of the efficiencies of adenine and cytosine base editors

(2020) Myungjae Song et al.   NATURE BIOTECHNOLOGY

Prediction of the sequence-specific cleavage activity of Cas9 variants

(2020) Nahye Kim et al.   NATURE BIOTECHNOLOGY

Predicting the efficiency of prime editing guide RNAs in human cells

(2020) Hui Kwon Kim et al.   NATURE BIOTECHNOLOGY

Unbiased investigation of specificities of prime editing systems in human cells

(2020) Do Yon Kim et al.   NUCLEIC ACIDS RESEARCH

Prime editing for functional repair in patient-derived disease models

(2020) Imre F. Schene et al.   Nature Communications

Restoration of visual function in adult mice with an inherited retinal disease via adenine base editing

(2020) Susie Suh et al.   Nature Biomedical Engineering

Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10

(2019) Morgan L. Maeder et al.   NATURE MEDICINE

Adeno-associated virus vector as a platform for gene therapy delivery

(2019) Dan Wang et al.   NATURE REVIEWS DRUG DISCOVERY

Adenine base editing in an adult mouse model of tyrosinaemia

(2019) Chun-Qing Song et al.   Nature Biomedical Engineering

Search-and-replace genome editing without double-strand breaks or donor DNA

(2019) Andrew V. Anzalone et al.   NATURE

SpCas9 activity prediction by DeepSpCas9, a deep learning–based model with high generalization performance

(2019) Hui Kwon Kim et al.   Science Advances

CRISPR-Cas9–mediated therapeutic editing of Rpe65 ameliorates the disease phenotypes in a mouse model of Leber congenital amaurosis

(2019) Dong Hyun Jo et al.   Science Advances

In vivo gene correction with targeted sequence substitution through microhomology-mediated end joining

(2018) Jeong Hong Shin et al.   BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS

Gene therapy and genome surgery in the retina

(2018) James E. DiCarlo et al.   JOURNAL OF CLINICAL INVESTIGATION

Adenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophy

(2018) Seuk-Min Ryu et al.   NATURE BIOTECHNOLOGY

The polyploid state restricts hepatocyte proliferation and liver regeneration

(2018) Patrick D Wilkinson et al.   HEPATOLOGY

Engineered CRISPR-Cas9 nuclease with expanded targeting space

(2018) Hiroshi Nishimasu et al.   SCIENCE

Effect of an intravitreal antisense oligonucleotide on vision in Leber congenital amaurosis due to a photoreceptor cilium defect

(2018) Artur V. Cideciyan et al.   NATURE MEDICINE

Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

(2017) Stephen Russell et al.   LANCET

Genetic interaction mapping in mammalian cells using CRISPR interference

(2017) Dan Du et al.   NATURE METHODS

Cas-analyzer: an online tool for assessing genome editing results using NGS data

(2016) Jeongbin Park et al.   BIOINFORMATICS

Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain

(2016) Benjamin E Deverman et al.   NATURE BIOTECHNOLOGY

Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo

(2016) Hao Yin et al.   NATURE BIOTECHNOLOGY

In vivo high-throughput profiling of CRISPR–Cpf1 activity

(2016) Hui K Kim et al.   NATURE METHODS

Digenome-seq: genome-wide profiling of CRISPR-Cas9 off-target effects in human cells

(2015) Daesik Kim et al.   NATURE METHODS

Long-Term Effect of Gene Therapy on Leber’s Congenital Amaurosis

(2015) James W.B. Bainbridge et al.   NEW ENGLAND JOURNAL OF MEDICINE

Improvement and Decline in Vision with Gene Therapy in Childhood Blindness

(2015) Samuel G. Jacobson et al.   NEW ENGLAND JOURNAL OF MEDICINE

Cas-OFFinder: a fast and versatile algorithm that searches for potential off-target sites of Cas9 RNA-guided endonucleases

(2014) S. Bae et al.   BIOINFORMATICS

Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype

(2014) Hao Yin et al.   NATURE BIOTECHNOLOGY

Surrogate reporter-based enrichment of cells containing RNA-guided Cas9 nuclease-induced mutations

(2014) Suresh Ramakrishna et al.   Nature Communications

Genome-Scale CRISPR-Cas9 Knockout Screening in Human Cells

(2013) Ophir Shalem et al.   SCIENCE

Spotlight on childhood blindness

(2011) José-Alain Sahel   JOURNAL OF CLINICAL INVESTIGATION

Leber congenital amaurosis due to RPE65 mutations and its treatment with gene therapy

(2010) Artur V. Cideciyan   PROGRESS IN RETINAL AND EYE RESEARCH

Adeno-associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo

(2009) Nicole K. Paulk et al.   HEPATOLOGY