Treatment of a metabolic liver disease by in vivo genome base editing in adult mice

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

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

Base editing with a Cpf1–cytidine deaminase fusion

(2018) Xiaosa Li et al.   NATURE BIOTECHNOLOGY

Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents

(2017) Xue Gao et al.   NATURE

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

(2017) Nicole M. Gaudelli et al.   NATURE

Correction of a pathogenic gene mutation in human embryos

(2017) Hong Ma et al.   NATURE

Increasing the genome-targeting scope and precision of base editing with engineered Cas9-cytidine deaminase fusions

(2017) Y Bill Kim et al.   NATURE BIOTECHNOLOGY

Engineered Cpf1 variants with altered PAM specificities

(2017) Linyi Gao et al.   NATURE BIOTECHNOLOGY

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

(2016) Alexis C. Komor et al.   NATURE

CrispRVariants charts the mutation spectrum of genome engineering experiments

(2016) Helen Lindsay et al.   NATURE BIOTECHNOLOGY

A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice

(2016) Yang Yang 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

Teleseismic S wave microseisms

(2016) K. Nishida et al.   SCIENCE

Crystal Structure of Staphylococcus aureus Cas9

(2015) Hiroshi Nishimasu et al.   CELL

Cpf1 Is a Single RNA-Guided Endonuclease of a Class 2 CRISPR-Cas System

(2015) Bernd Zetsche et al.   CELL

Engineered CRISPR-Cas9 nucleases with altered PAM specificities

(2015) Benjamin P. Kleinstiver et al.   NATURE

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

Broadening the targeting range of Staphylococcus aureus CRISPR-Cas9 by modifying PAM recognition

(2015) Benjamin P Kleinstiver et al.   NATURE BIOTECHNOLOGY

Development of an intein-mediated split–Cas9 system for gene therapy

(2015) Dong-Jiunn Jeffery Truong et al.   NUCLEIC ACIDS RESEARCH

Computationally designed liver-specific transcriptional modules and hyperactive factor IX improve hepatic gene therapy

(2014) N. Nair et al.   BLOOD

Crystal Structure of Cas9 in Complex with Guide RNA and Target DNA

(2014) Hiroshi Nishimasu et al.   CELL

Treatment of phenylketonuria using minicircle-based naked-DNA gene transfer to murine liver

(2014) Hiu Man Viecelli et al.   HEPATOLOGY

Phenotypic Reversion of Fair Hair upon Gene Therapy of the Phenylketonuria Mice

(2014) Beat Thöny et al.   HUMAN GENE THERAPY

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

(2014) Hao Yin et al.   NATURE BIOTECHNOLOGY

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

(2013) J. Zhang et al.   BIOINFORMATICS

Redundant Sources of Wnt Regulate Intestinal Stem Cells and Promote Formation of Paneth Cells

(2012) Henner F. Farin et al.   GASTROENTEROLOGY

Quantification of phenylalanine hydroxylase activity by isotope-dilution liquid chromatography–electrospray ionization tandem mass spectrometry

(2012) Caroline Heintz et al.   MOLECULAR GENETICS AND METABOLISM

DNA repair by nonhomologous end joining and homologous recombination during cell cycle in human cells

(2011) Zhiyong Mao et al.   CELL CYCLE

Phenylalanine hydroxylase deficiency

(2011) John J. Mitchell et al.   GENETICS IN MEDICINE

Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges

(2011) Federico Mingozzi et al.   NATURE REVIEWS GENETICS

State-of-the-art gene-based therapies: the road ahead

(2011) Mark A. Kay   NATURE REVIEWS GENETICS

Lgr5+ve Stem Cells Drive Self-Renewal in the Stomach and Build Long-Lived Gastric Units In Vitro

(2010) Nick Barker et al.   Cell Stem Cell

Phenylketonuria

(2010) Nenad Blau et al.   LANCET

Animal models of brain dysfunction in phenylketonuria☆

(2009) A.E. Martynyuk et al.   MOLECULAR GENETICS AND METABOLISM

Comparison of nonhomologous end joining and homologous recombination in human cells

(2008) Z MAO et al.   DNA REPAIR