4.8 Article

Programmable RNA base editing with a single gRNA-free enzyme

相关参考文献

注意:仅列出部分参考文献,下载原文获取全部文献信息。
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 Cell Biology

Eliminating base-editor-induced genome-wide and transcriptome-wide off-target mutations

Lijie Wang et al.

Summary: The study introduces a novel base editor system, tBE, which efficiently performs C-to-T editing by fusing a cleavable dCDI domain, reducing off-target mutations. The tBE system remains inactive at off-target sites, preventing unintended mutations, and activates for precise base editing at on-target sites.

NATURE CELL BIOLOGY (2021)

Article Biotechnology & Applied Microbiology

Evaluation and minimization of Cas9-independent off-target DNA editing by cytosine base editors

Jordan L. Doman et al.

NATURE BIOTECHNOLOGY (2020)

Editorial Material Biotechnology & Applied Microbiology

Immunity to Cas9 as an Obstacle to Persistent Genome Editing

Veronica Gough et al.

MOLECULAR THERAPY (2020)

Article Biochemistry & Molecular Biology

Programmable C-to-U RNA editing using the human APOBEC3A deaminase

Xinxin Huang et al.

EMBO JOURNAL (2020)

Article Biochemistry & Molecular Biology

REPAIRx, a specific yet highly efficient programmable A > I RNA base editor

Yajing Liu et al.

EMBO JOURNAL (2020)

Article Chemistry, Multidisciplinary

Programmable RNA methylation and demethylation using PUF RNA binding proteins

Kouki Shinoda et al.

CHEMICAL COMMUNICATIONS (2020)

Article Biotechnology & Applied Microbiology

Precise RNA editing by recruiting endogenous ADARs with antisense oligonucleotides

Tobias Merkle et al.

NATURE BIOTECHNOLOGY (2019)

Article Biochemistry & Molecular Biology

Identification of preexisting adaptive immunity to Cas9 proteins in humans

Carsten T. Charlesworth et al.

NATURE MEDICINE (2019)

Article Biochemical Research Methods

In vivo RNA editing of point mutations via RNA-guided adenosine deaminases

Dhruva Katrekar et al.

NATURE METHODS (2019)

Article Biochemistry & Molecular Biology

A Quantitative and Predictive Model for RNA Binding by Human Pumilio Proteins

Inga Jarmoskaite et al.

MOLECULAR CELL (2019)

Article Multidisciplinary Sciences

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

Julian Grunewald et al.

NATURE (2019)

Article Multidisciplinary Sciences

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

Changyang Zhou et al.

NATURE (2019)

Article Biotechnology & Applied Microbiology

Programmable RNA editing by recruiting endogenous ADAR using engineered RNAs

Liang Qu et al.

NATURE BIOTECHNOLOGY (2019)

Article Multidisciplinary Sciences

A cytosine deaminase for programmable single-base RNA editing

Omar O. Abudayyeh et al.

SCIENCE (2019)

Article Biochemical Research Methods

Elucidating the editome: bioinformatics approaches for RNA editing detection

Maria Angela Diroma et al.

BRIEFINGS IN BIOINFORMATICS (2019)

Article Biochemical Research Methods

Efficient and precise editing of endogenous transcripts with SNAP-tagged ADARs

Paul Vogel et al.

NATURE METHODS (2018)

Article Biochemistry & Molecular Biology

Expanding RNA binding specificity and affinity of engineered PUF domains

Yang-Yang Zhao et al.

NUCLEIC ACIDS RESEARCH (2018)

Article Biotechnology & Applied Microbiology

Efficient base editing in methylated regions with a human APOBEC3A-Cas9 fusion

Xiao Wang et al.

NATURE BIOTECHNOLOGY (2018)

Article Biotechnology & Applied Microbiology

An APOBEC3A-Cas9 base editor with minimized bystander and off-target activities

Jason M. Gehrke et al.

NATURE BIOTECHNOLOGY (2018)

Article Biotechnology & Applied Microbiology

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

Daesik Kim et al.

NATURE BIOTECHNOLOGY (2017)

Article Multidisciplinary Sciences

RNA editing with CRISPR-Cas13

David B. T. Cox et al.

SCIENCE (2017)

Article Multidisciplinary Sciences

Construction of a guide-RNA for site-directed RNA mutagenesis utilising intracellular A-to-I RNA editing

Masatora Fukuda et al.

Scientific Reports (2017)

Article Multidisciplinary Sciences

Stem-loop structure preference for site-specific RNA editing by APOBEC3A and APOBEC3G

Shraddha Sharma et al.

Article Biochemical Research Methods

ggseqlogo: a versatile R package for drawing sequence logos

Omar Wagih

BIOINFORMATICS (2017)

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 Biochemistry & Molecular Biology

Structures of human ADAR2 bound to dsRNA reveal base-flipping mechanism and basis for site selectivity

Melissa M. Matthews et al.

NATURE STRUCTURAL & MOLECULAR BIOLOGY (2016)

Article Biochemistry & Molecular Biology

Kinetics of the CRISPR-Cas9 effector complex assembly and the role of 3'-terminal segment of guide RNA

Vladimir Mekler et al.

NUCLEIC ACIDS RESEARCH (2016)

Article Multidisciplinary Sciences

Programmable RNA-binding protein composed of repeats of a single modular unit

Katarzyna P. Adamala et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2016)

Review Biochemistry & Molecular Biology

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

Jason D. Salter et al.

TRENDS IN BIOCHEMICAL SCIENCES (2016)

Article Biochemistry & Molecular Biology

Structures of human ADAR2 bound to dsRNA reveal base-flipping mechanism and basis for site selectivity

Melissa M. Matthews et al.

NATURE STRUCTURAL & MOLECULAR BIOLOGY (2016)

Article Biochemistry & Molecular Biology

A Phenotypic Screen for Functional Mutants of Human Adenosine Deaminase Acting on RNA 1

Yuru Wang et al.

ACS CHEMICAL BIOLOGY (2015)

Article Biochemistry & Molecular Biology

The ssDNA Mutator APOBEC3A Is Regulated by Cooperative Dimerization

Markus-Frederik Bohn et al.

STRUCTURE (2015)

Review Cell Biology

Engineering RNA-binding proteins with diverse activities

Huanhuan Wei et al.

WILEY INTERDISCIPLINARY REVIEWS-RNA (2015)

Article Biochemistry & Molecular Biology

A-to-I RNA editing occurs at over a hundred million genomic sites, located in a majority of human genes

Lily Bazak et al.

GENOME RESEARCH (2014)

Article Biochemical Research Methods

STAR: ultrafast universal RNA-seq aligner

Alexander Dobin et al.

BIOINFORMATICS (2013)

Article Multidisciplinary Sciences

Correction of mutations within the cystic fibrosis transmembrane conductance regulator by site-directed RNA editing

Maria Fernanda Montiel-Gonzalez et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2013)

Article Chemistry, Multidisciplinary

An RNA-Deaminase Conjugate Selectively Repairs Point Mutations

Thorsten Stafforst et al.

ANGEWANDTE CHEMIE-INTERNATIONAL EDITION (2012)

Article Biochemistry & Molecular Biology

Accurate identification of A-to-I RNA editing in human by transcriptome sequencing

Jae Hoon Bahn et al.

GENOME RESEARCH (2012)

Article Multidisciplinary Sciences

Mechanistic insights into editing-site specificity of ADARs

Ashani Kuttan et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2012)

Article Multidisciplinary Sciences

Engineering RNA endonucleases with customized sequence specificities

Rajarshi Choudhury et al.

NATURE COMMUNICATIONS (2012)

Article Biochemistry & Molecular Biology

Specific and Modular Binding Code for Cytosine Recognition in Pumilio/FBF (PUF) RNA-binding Domains

Shuyun Dong et al.

JOURNAL OF BIOLOGICAL CHEMISTRY (2011)

Article Biochemistry & Molecular Biology

A universal code for RNA recognition by PUF proteins

Aleksandra Filipovska et al.

NATURE CHEMICAL BIOLOGY (2011)

Article Multidisciplinary Sciences

Targeted translational regulation using the PUF protein family scaffold

Amy Cooke et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2011)

Review Biochemistry & Molecular Biology

Functions and Regulation of RNA Editing by ADAR Deaminases

Kazuko Nishikura

ANNUAL REVIEW OF BIOCHEMISTRY, VOL 79 (2010)

Article Biochemical Research Methods

The Sequence Alignment/Map format and SAMtools

Heng Li et al.

BIOINFORMATICS (2009)

Article Biochemical Research Methods

Fast and accurate short read alignment with Burrows-Wheeler transform

Heng Li et al.

BIOINFORMATICS (2009)

Article Biochemical Research Methods

Engineering splicing factors with designed specificities

Yang Wang et al.

NATURE METHODS (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 Biochemical Research Methods

Imaging dynamics of endogenous mitochondrial RNA in single living cells

Takeaki Ozawa et al.

NATURE METHODS (2007)

Article Multidisciplinary Sciences

Engineering RNA sequence specificity of Pumilio repeats

Cheorn-Gil Cheong et al.

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

Article Biochemistry & Molecular Biology

Double-stranded RNA adenosine deaminases ADAR1 and ADAR2 have overlapping specificities

KA Lehmann et al.

BIOCHEMISTRY (2000)