In vivo base editing restores sensory transduction and transiently improves auditory function in a mouse model of recessive deafness

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

Improved TMC1 gene therapy restores hearing and balance in mice with genetic inner ear disorders

(2019) Carl A. Nist-Lund et al.   Nature Communications

AAV2.7m8 is a powerful viral vector for inner ear gene therapy

(2019) Kevin Isgrig et al.   Nature Communications

Cytosine, but not adenine, base editors induce genome-wide off-target mutations in rice

(2019) Shuai Jin et al.   SCIENCE

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

Off-target RNA mutation induced by DNA base editing and its elimination by mutagenesis

(2019) Changyang Zhou et al.   NATURE

Continuous evolution of base editors with expanded target compatibility and improved activity

(2019) Benjamin W. Thuronyi et al.   NATURE BIOTECHNOLOGY

Allele-specific gene editing prevents deafness in a model of dominant progressive hearing loss

(2019) Bence György et al.   NATURE MEDICINE

Off-Target Editing by CRISPR-Guided DNA Base Editors

(2019) SeHee Park et al.   BIOCHEMISTRY

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

(2018) Luke W Koblan et al.   NATURE BIOTECHNOLOGY

Identification of four TMC1 variations in different Chinese families with hereditary hearing loss

(2018) Hongyang Wang et al.   Molecular Genetics & Genomic Medicine

Evolved Cas9 variants with broad PAM compatibility and high DNA specificity

(2018) Johnny H. Hu et al.   NATURE

TMC1 Forms the Pore of Mechanosensory Transduction Channels in Vertebrate Inner Ear Hair Cells

(2018) Bifeng Pan et al.   NEURON

Engineered CRISPR-Cas9 nuclease with expanded targeting space

(2018) Hiroshi Nishimasu et al.   SCIENCE

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

(2018) Lukas Villiger et al.   NATURE MEDICINE

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

(2018) Holly A. Rees et al.   NATURE REVIEWS GENETICS

Entering the Modern Era of Gene Therapy

(2018) Xavier M. Anguela et al.   Annual Review of Medicine

Emerging Gene Therapies for Genetic Hearing Loss

(2017) Hena Ahmed et al.   JARO-JOURNAL OF THE ASSOCIATION FOR RESEARCH IN OTOLARYNGOLOGY

Rescue of Outer Hair Cells with Antisense Oligonucleotides in Usher Mice Is Dependent on Age of Treatment

(2017) Abhilash Ponnath et al.   JARO-JOURNAL OF THE ASSOCIATION FOR RESEARCH IN OTOLARYNGOLOGY

Biochemical Regulatory Features of Activation-Induced Cytidine Deaminase Remain Conserved from Lampreys to Humans

(2017) Emma M. Quinlan et al.   MOLECULAR AND CELLULAR BIOLOGY

Gene Therapy Restores Balance and Auditory Functions in a Mouse Model of Usher Syndrome

(2017) Kevin Isgrig et al.   MOLECULAR THERAPY

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

Gene therapy restores auditory and vestibular function in a mouse model of Usher syndrome type 1c

(2017) Bifeng Pan et al.   NATURE BIOTECHNOLOGY

A synthetic AAV vector enables safe and efficient gene transfer to the mammalian inner ear

(2017) Lukas D Landegger et al.   NATURE BIOTECHNOLOGY

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

(2017) Y Bill Kim et al.   NATURE BIOTECHNOLOGY

Genome-wide target specificities of CRISPR RNA-guided programmable deaminases

(2017) Daesik Kim et al.   NATURE BIOTECHNOLOGY

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

(2017) Shengdar Q Tsai et al.   NATURE METHODS

Congenital hearing loss

(2017) Anna M. H. Korver et al.   Nature Reviews Disease Primers

Improved base excision repair inhibition and bacteriophage Mu Gam protein yields C:G-to-T:A base editors with higher efficiency and product purity

(2017) Alexis C. Komor et al.   Science Advances

Cochlear gene therapy with ancestral AAV in adult mice: complete transduction of inner hair cells without cochlear dysfunction

(2017) Jun Suzuki et al.   Scientific Reports

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

(2016) Alexis C. Komor et al.   NATURE

High-fidelity CRISPR–Cas9 nucleases with no detectable genome-wide off-target effects

(2016) Benjamin P. Kleinstiver et al.   NATURE

Recessive mutations of TMC1 associated with moderate to severe hearing loss

(2016) Ayesha Imtiaz et al.   NEUROGENETICS

Targeted nucleotide editing using hybrid prokaryotic and vertebrate adaptive immune systems

(2016) K. Nishida et al.   SCIENCE

The APOBEC Protein Family: United by Structure, Divergent in Function

(2016) Jason D. Salter et al.   TRENDS IN BIOCHEMICAL SCIENCES

Progressive Hearing Loss in Mice Carrying a Mutation in Usp53

(2015) Marcin Kazmierczak et al.   JOURNAL OF NEUROSCIENCE

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

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

ClinVar: public archive of interpretations of clinically relevant variants

(2015) Melissa J. Landrum et al.   NUCLEIC ACIDS RESEARCH

Tmc gene therapy restores auditory function in deaf mice

(2015) Charles Askew et al.   Science Translational Medicine

TMC1 and TMC2 Localize at the Site of Mechanotransduction in Mammalian Inner Ear Hair Cell Stereocilia

(2015) Kiyoto Kurima et al.   Cell Reports

The Status ofRPE65Gene Therapy Trials: Safety and Efficacy

(2015) Eric A. Pierce et al.   Cold Spring Harbor Perspectives in Medicine

Gene therapy on the move

(2013) Kerstin B. Kaufmann et al.   EMBO Molecular Medicine

Tricellulin deficiency affects tight junction architecture and cochlear hair cells

(2013) Gowri Nayak et al.   JOURNAL OF CLINICAL INVESTIGATION

Rescue of hearing and vestibular function by antisense oligonucleotides in a mouse model of human deafness

(2013) Jennifer J Lentz et al.   NATURE MEDICINE

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

(2013) Melissa J. Landrum et al.   NUCLEIC ACIDS RESEARCH

Nucleic acid determinants for selective deamination of DNA over RNA by activation-induced deaminase

(2013) C. S. Nabel et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Identification of Three Novel Hearing Loss Mouse Strains with Mutations in the Tmc1 Gene

(2012) Shehnaaz S.M. Manji et al.   AMERICAN JOURNAL OF PATHOLOGY

Restoration of Hearing in the VGLUT3 Knockout Mouse Using Virally Mediated Gene Therapy

(2012) Omar Akil et al.   NEURON

Mechanotransduction in mouse inner ear hair cells requires transmembrane channel–like genes

(2011) Yoshiyuki Kawashima et al.   JOURNAL OF CLINICAL INVESTIGATION

Transcriptome-wide sequencing reveals numerous APOBEC1 mRNA-editing targets in transcript 3′ UTRs

(2011) Brad R Rosenberg et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

The Complex and Evolving Story of T cell Activation to AAV Vector-encoded Transgene Products

(2010) Lauren E Mays et al.   MOLECULAR THERAPY

Function and Expression Pattern of Nonsyndromic Deafness Genes

(2009) Nele Hilgert et al.   CURRENT MOLECULAR MEDICINE

Mutations in TMC1 contribute significantly to nonsyndromic autosomal recessive sensorineural hearing loss: A report of five novel mutations

(2009) Aslı Sırmacı et al.   INTERNATIONAL JOURNAL OF PEDIATRIC OTORHINOLARYNGOLOGY

A Portable Hot Spot Recognition Loop Transfers Sequence Preferences from APOBEC Family Members to Activation-induced Cytidine Deaminase

(2009) Rahul M. Kohli et al.   JOURNAL OF BIOLOGICAL CHEMISTRY