4.6 Article

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

Related references

Note: Only part of the references are listed.
Article Biochemistry & Molecular Biology

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

Morgan L. Maeder et al.

NATURE MEDICINE (2019)

Article Biochemistry & Molecular Biology

Nephron segment-specific gene expression using AAV vectors

Laureano D. Asico et al.

BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS (2018)

Review Biotechnology & Applied Microbiology

Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy

Dongsheng Duan

MOLECULAR THERAPY (2018)

Letter Biotechnology & Applied Microbiology

The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice

Juliette Hordeaux et al.

MOLECULAR THERAPY (2018)

Article Biotechnology & Applied Microbiology

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

Seuk-Min Ryu et al.

NATURE BIOTECHNOLOGY (2018)

Article Biotechnology & Applied Microbiology

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

Luke W. Koblan et al.

NATURE BIOTECHNOLOGY (2018)

Article Medicine, General & Internal

Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

E. Mercuri et al.

NEW ENGLAND JOURNAL OF MEDICINE (2018)

Article Biochemistry & Molecular Biology

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

Lukas Villiger et al.

NATURE MEDICINE (2018)

Review Genetics & Heredity

Base editing: precision chemistry on the genome and transcriptome of living cells

Holly A. Rees et al.

NATURE REVIEWS GENETICS (2018)

Review Neurosciences

Purkinje Cell Signaling Deficits in Animal Models of Ataxia

Eriola Hoxha et al.

FRONTIERS IN SYNAPTIC NEUROSCIENCE (2018)

Article Biochemistry & Molecular Biology

AAV9-NPC1 significantly ameliorates Purkinje cell death and behavioral abnormalities in mouse NPC disease

Chang Xie et al.

JOURNAL OF LIPID RESEARCH (2017)

Article Multidisciplinary Sciences

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

Nicole M. Gaudelli et al.

NATURE (2017)

Article Biotechnology & Applied Microbiology

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

Y. Bill Kim et al.

NATURE BIOTECHNOLOGY (2017)

Article Biochemical Research Methods

Massively parallel single-nucleus RNA-seq with DroNc-seq

Naomi Habib et al.

NATURE METHODS (2017)

Article Biochemical Research Methods

CIRCLE-seq: a highly sensitive in vitro screen for genome-wide CRISPR Cas9 nuclease off-targets

Shengdar Q. Tsai et al.

NATURE METHODS (2017)

Article Medicine, General & Internal

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

J. R. Mendell et al.

NEW ENGLAND JOURNAL OF MEDICINE (2017)

Article Multidisciplinary Sciences

Genetic rescue models refute nonautonomous rod cell death in retinitis pigmentosa

Susanne F. Koch et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2017)

Article Neurosciences

Evaluating Efficiencies of Dual AAV Approaches for Retinal Targeting

Livia S. Carvalho et al.

FRONTIERS IN NEUROSCIENCE (2017)

Article Hematology

In Vivo Base Editing of PCSK9 (Proprotein Convertase Subtilisin/Kexin Type 9) as a Therapeutic Alternative to Genome Editing

Alexandra C. Chadwick et al.

ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY (2017)

Article Chemistry, Multidisciplinary

Design of a Split Intein with Exceptional Protein Splicing Activity

Adam J. Stevens et al.

JOURNAL OF THE AMERICAN CHEMICAL SOCIETY (2016)

Article Multidisciplinary Sciences

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

Alexis C. Komor et al.

NATURE (2016)

Article Biotechnology & Applied Microbiology

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

Benjamin E. Deverman et al.

NATURE BIOTECHNOLOGY (2016)

Article Biotechnology & Applied Microbiology

Evaluation of off-target and on-target scoring algorithms and integration into the guide RNA selection tool CRISPOR

Maximilian Haeussler et al.

GENOME BIOLOGY (2016)

Article Multidisciplinary Sciences

In vivo genome editing using Staphylococcus aureus Cas9

F. Ann Ran et al.

NATURE (2015)

Letter Biotechnology & Applied Microbiology

A split-Cas9 architecture for inducible genome editing and transcription modulation

Bernd Zetsche et al.

NATURE BIOTECHNOLOGY (2015)

Article Biochemistry & Molecular Biology

Small molecule-triggered Cas9 protein with improved genome-editing specificity

Kevin M. Davis et al.

NATURE CHEMICAL BIOLOGY (2015)

Article Biochemistry & Molecular Biology

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

Dong-Jiunn Jeffery Truong et al.

NUCLEIC ACIDS RESEARCH (2015)

Article Multidisciplinary Sciences

Rational design of a split-Cas9 enzyme complex

Addison V. Wright et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2015)

Article Biotechnology & Applied Microbiology

In vivo interrogation of gene function in the mammalian brain using CRISPR-Cas9

Lukasz Swiech et al.

NATURE BIOTECHNOLOGY (2015)

Article Biotechnology & Applied Microbiology

Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo

John A. Zuris et al.

NATURE BIOTECHNOLOGY (2015)

Article Medicine, General & Internal

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

A. C. Nathwani et al.

NEW ENGLAND JOURNAL OF MEDICINE (2014)

Article Biochemistry & Molecular Biology

ClinVar: public archive of relationships among sequence variation and human phenotype

Melissa J. Landrum et al.

NUCLEIC ACIDS RESEARCH (2014)

Article Chemistry, Multidisciplinary

Extein Residues Play an Intimate Role in the Rate-Limiting Step of Protein Trans-Splicing

Neel H. Shah et al.

JOURNAL OF THE AMERICAN CHEMICAL SOCIETY (2013)

Article Multidisciplinary Sciences

Capsid Serotype and Timing of Injection Determines AAV Transduction in the Neonatal Mice Brain

Paramita Chakrabarty et al.

PLOS ONE (2013)

Article Neurosciences

Anatomically Defined Neuron-Based Rescue of Neurodegenerative Niemann-Pick Type C Disorder

Manuel E. Lopez et al.

JOURNAL OF NEUROSCIENCE (2011)

Article Biochemistry & Molecular Biology

Conditional Niemann-Pick C mice demonstrate cell autonomous Purkinje cell neurodegeneration

Matthew J. Elrick et al.

HUMAN MOLECULAR GENETICS (2010)

Article Biotechnology & Applied Microbiology

Effect of Genome Size on AAV Vector Packaging

Zhijian Wu et al.

MOLECULAR THERAPY (2010)

Article Biotechnology & Applied Microbiology

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

Kevin D. Foust et al.

NATURE BIOTECHNOLOGY (2009)

Article Biotechnology & Applied Microbiology

Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection

Carmela Zincarelli et al.

MOLECULAR THERAPY (2008)

Article Multidisciplinary Sciences

Pregnane X receptor (PXR) activation: A mechanism for neuroprotection in a mouse model of Niemann-Pick C disease

S. Joshua Langmade et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2006)

Article Biotechnology & Applied Microbiology

Robust systemic transduction with AAV9 vectors in mice: Efficient global cardiac gene transfer superior to that of AAV8

Katsuya Inagaki et al.

MOLECULAR THERAPY (2006)

Review Biotechnology & Applied Microbiology

Adeno-associated virus serotypes: Vector toolkit for human gene therapy

Zhijian Wu et al.

MOLECULAR THERAPY (2006)

Article Biotechnology & Applied Microbiology

CellProfiler: image analysis software for identifying and quantifying cell phenotypes

Anne E. Carpenter et al.

GENOME BIOLOGY (2006)

Correction Genetics & Heredity

Uncoupling of longevity and telomere length in C-elegans (vol 1, pg 295, 2005)

M Raices et al.

PLOS GENETICS (2005)

Article Biotechnology & Applied Microbiology

Low therapeutic threshold for hepatocyte replacement in murine phenylketonuria

K Hamman et al.

MOLECULAR THERAPY (2005)

Article Biochemistry & Molecular Biology

Rescue of neurodegeneration in Niemann-Pick C mice by a prion-promoter-driven Npc1 cDNA transgene

SK Loftus et al.

HUMAN MOLECULAR GENETICS (2002)

Article Genetics & Heredity

Mutations and polymorphisms in the human ornithine transcarbamylase gene

M Tuchman et al.

HUMAN MUTATION (2002)

Article Biotechnology & Applied Microbiology

Expanding AAV packaging capacity with trans-splicing or overlapping vectors:: A quantitative comparison

DS Duan et al.

MOLECULAR THERAPY (2001)