4.8 Article

Template-independent genome editing in the Pcdh15av-3j mouse, a model of human DFNB23 nonsyndromic deafness

Related references

Note: Only part of the references are listed.
Article Multidisciplinary Sciences

In vivo base editing rescues Hutchinson-Gilford progeria syndrome in mice

Luke W. Koblan et al.

Summary: Using adenine base editor (ABE), researchers successfully corrected the pathogenic mutation in fibroblasts from children with Hutchinson-Gilford progeria syndrome, mitigating RNA mis-splicing, reducing progerin levels, and correcting nuclear abnormalities. In vivo base editing with AAV9 encoding ABE in transgenic mice resulted in substantial, durable correction of the mutation, restoration of normal RNA splicing, and improvement of vascular pathology, ultimately extending the lifespan of the mice. These findings demonstrate the potential of in vivo base editing as a treatment for genetic diseases.

NATURE (2021)

Article Multidisciplinary Sciences

Unconstrained genome targeting with near-PAMless engineered CRISPR-Cas9 variants

Russell T. Walton et al.

SCIENCE (2020)

Letter Biotechnology & Applied Microbiology

CRISPResso2 provides accurate and rapid genome editing sequence analysis

Kendell Clement et al.

NATURE BIOTECHNOLOGY (2019)

Article Multidisciplinary Sciences

Dual AAV-mediated gene therapy restores hearing in a DFNB9 mouse model

Omar Akil et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2019)

Article Biotechnology & Applied Microbiology

Large dataset enables prediction of repair after CRISPR-Cas9 editing in primary T cells

Ryan T. Leenay et al.

NATURE BIOTECHNOLOGY (2019)

Article Biochemistry & Molecular Biology

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

Bence Gyorgy et al.

NATURE MEDICINE (2019)

Article Biochemistry & Molecular Biology

Target-Specific Precision of CRISPR-Mediated Genome Editing

Anob M. Chakrabarti et al.

MOLECULAR CELL (2019)

Article Biotechnology & Applied Microbiology

Predicting the mutations generated by repair of Cas9-induced double-strand breaks

Felicity Allen et al.

NATURE BIOTECHNOLOGY (2019)

Article Multidisciplinary Sciences

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

Carl A. Nist-Lund et al.

NATURE COMMUNICATIONS (2019)

Article Multidisciplinary Sciences

Predictable and precise template-free CRISPR editing of pathogenic variants

Max W. Shen et al.

NATURE (2018)

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

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

Bifeng Pan et al.

NATURE BIOTECHNOLOGY (2017)

Review Biochemistry & Molecular Biology

Refining strategies to translate genome editing to the clinic

Tatjana I. Cornu et al.

NATURE MEDICINE (2017)

Article Multidisciplinary Sciences

Local gene therapy durably restores vestibular function in a mouse model of Usher syndrome type 1G

Alice Emptoz et al.

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

Article Biochemistry & Molecular Biology

DNA Repair Profiling Reveals Nonrandom Outcomes at Cas9-Mediated Breaks

Megan van Overbeek et al.

MOLECULAR CELL (2016)

Article Multidisciplinary Sciences

In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration

Keiichiro Suzuki et al.

NATURE (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)

Review Biochemistry & Molecular Biology

Therapeutic genome editing: prospects and challenges

David Benjamin Turitz Cox et al.

NATURE MEDICINE (2015)

Article Multidisciplinary Sciences

CCTop: An Intuitive, Flexible and Reliable CRISPR/Cas9 Target Prediction Tool

Manuel Stemmer et al.

PLOS ONE (2015)

Article Biochemistry & Molecular Biology

CRISPR-Cas9 Knockin Mice for Genome Editing and Cancer Modeling

Randall J. Platt et al.

Article Biotechnology & Applied Microbiology

Genome-wide recessive genetic screening in mammalian cells with a lentiviral CRISPR-guide RNA library

Hiroko Koike-Yusa et al.

NATURE BIOTECHNOLOGY (2014)

Article Biotechnology & Applied Microbiology

Improving CRISPR-Cas nuclease specificity using truncated guide RNAs

Yanfang Fu et al.

NATURE BIOTECHNOLOGY (2014)

Article Biochemical Research Methods

Using injectoporation to deliver genes to mechanosensory hair cells

Wei Xiong et al.

NATURE PROTOCOLS (2014)

Article Biochemical Research Methods

Genome engineering using the CRISPR-Cas9 system

F. Ann Ran et al.

NATURE PROTOCOLS (2013)

Article Biochemical Research Methods

NIH Image to ImageJ: 25 years of image analysis

Caroline A. Schneider et al.

NATURE METHODS (2012)

Review Genetics & Heredity

Double-Strand Break End Resection and Repair Pathway Choice

Lorraine S. Symington et al.

ANNUAL REVIEW OF GENETICS, VOL 45 (2011)

Article Multidisciplinary Sciences

Cadherin 23 and protocadherin 15 interact to form tip-link filaments in sensory hair cells

Piotr Kazmierczak et al.

NATURE (2007)

Article Neurosciences

Progression of inner ear pathology in Ames waltzer mice and the role of protocadherin 15 in hair cell development

Karen S. Pawlowski et al.

JARO-JOURNAL OF THE ASSOCIATION FOR RESEARCH IN OTOLARYNGOLOGY (2006)

Article Genetics & Heredity

Human gene mutation database (HGMD®):: 2003 update

PD Stenson et al.

HUMAN MUTATION (2003)

Article Developmental Biology

Expression of Pcdh15 in the inner ear, nervous system and various epithelia of the developing embryo

CL Murcia et al.

MECHANISMS OF DEVELOPMENT (2001)

Article Biochemical Research Methods

A comparison of video and magnetic search coil recordings of mouse eye movements

JS Stahl et al.

JOURNAL OF NEUROSCIENCE METHODS (2000)