Enhanced genome editing to ameliorate a genetic metabolic liver disease through co-delivery of adeno-associated virus receptor

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

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

Transcriptome-wide off-target RNA editing induced by CRISPR-guided DNA base editors

(2019) Julian Grünewald et al.   NATURE

Analysis and minimization of cellular RNA editing by DNA adenine base editors

(2019) Holly A. Rees et al.   Science Advances

Cas9-nickase–mediated genome editing corrects hereditary tyrosinemia in rats

(2018) Yanjiao Shao et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Gene therapy comes of age

(2018) Cynthia E. Dunbar et al.   SCIENCE

Nucleosomes inhibit target cleavage by CRISPR-Cas9 in vivo

(2018) Robert M. Yarrington et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

(2018) Lukas Villiger 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

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

CRISPR/Cas9-mediated gene knockout is insensitive to target copy number but is dependent on guide RNA potency and Cas9/sgRNA threshold expression level

(2017) Garmen Yuen et al.   NUCLEIC ACIDS RESEARCH

The complete European guidelines on phenylketonuria: diagnosis and treatment

(2017) A. M. J. van Wegberg et al.   Orphanet Journal of Rare Diseases

The Chromatin Structure Differentially Impacts High-Specificity CRISPR-Cas9 Nuclease Strategies

(2017) Xiaoyu Chen et al.   Molecular Therapy-Nucleic Acids

Low-Dose Gene Therapy for Murine PKU Using Episomal Naked DNA Vectors Expressing PAH from Its Endogenous Liver Promoter

(2017) Hiu Man Grisch-Chan et al.   Molecular Therapy-Nucleic Acids

CRISPR/Cas9‐mediated somatic correction of a novel coagulator factor IX gene mutation ameliorates hemophilia in mouse

(2016) Yuting Guan et al.   EMBO Molecular Medicine

An essential receptor for adeno-associated virus infection

(2016) S. Pillay et al.   NATURE

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

(2016) Alexis C. Komor et al.   NATURE

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

A universal system to select gene-modified hepatocytes in vivo

(2016) Sean Nygaard et al.   Science Translational Medicine

Systemic delivery of adeno-associated viral vectors

(2016) Dongsheng Duan   Current Opinion in Virology

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

Large genomic fragment deletion and functional gene cassette knock-in via Cas9 protein mediated genome editing in one-cell rodent embryos

(2015) Liren Wang et al.   Scientific Reports

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

(2014) Hiu Man Viecelli et al.   HEPATOLOGY

Promoterless gene targeting without nucleases ameliorates haemophilia B in mice

(2014) A. Barzel et al.   NATURE

CRISPR/Cas-mediated genome editing in the rat via direct injection of one-cell embryos

(2014) Yanjiao Shao et al.   Nature Protocols

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

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

Hepatic Gene Transfer in Neonatal Mice by Adeno-Associated Virus Serotype 8 Vector

(2011) Lili Wang et al.   HUMAN GENE THERAPY

Complete restoration of phenylalanine oxidation in phenylketonuria mouse by a self-complementary adeno-associated virus vector

(2011) Hiroya Yagi et al.   JOURNAL OF GENE MEDICINE

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

(2011) Hojun Li et al.   NATURE

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

(2011) Federico Mingozzi et al.   NATURE REVIEWS GENETICS

Phenylketonuria

(2010) Nenad Blau et al.   LANCET

PKU—What is daily practice in various centres in Europe?

(2009) F. J. van Spronsen et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Management of phenylketonuria in Europe: Survey results from 19 countries

(2009) Nenad Blau et al.   MOLECULAR GENETICS AND METABOLISM

Correction of Murine PKU Following AAV-mediated Intramuscular Expression of a Complete Phenylalanine Hydroxylating System

(2008) Zhaobing Ding et al.   MOLECULAR THERAPY

Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection

(2008) Carmela Zincarelli et al.   MOLECULAR THERAPY

Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at lower doses

(2008) L. Zhong et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA