Related references
Note: Only part of the references are listed.Efficient in vivo genome editing prevents hypertrophic cardiomyopathy in mice
Daniel Reichart et al.
NATURE MEDICINE (2023)
Gene Therapy Advances: A Meta-Analysis of AAV Usage in Clinical Settings
Hau Kiu Edna Au et al.
FRONTIERS IN MEDICINE (2022)
RBM20S639G mutation is a high genetic risk factor for premature death through RNA-protein condensates
Chunyan Wang et al.
JOURNAL OF MOLECULAR AND CELLULAR CARDIOLOGY (2022)
Precise genomic editing of pathogenic mutations in RBM20 rescues dilated cardiomyopathy
Takahiko Nishiyama et al.
SCIENCE TRANSLATIONAL MEDICINE (2022)
Base editors hit the clinic
Katie Kingwell
NATURE REVIEWS DRUG DISCOVERY (2022)
Pathogenic variants damage cell composition and single-cell transcription in cardiomyopathies
Daniel Reichart et al.
SCIENCE (2022)
Immunogenicity and toxicity of AAV gene therapy
Hildegund C. J. Ertl
FRONTIERS IN IMMUNOLOGY (2022)
The Role of Recombinant AAV in Precise Genome Editing
Swati Bijlani et al.
FRONTIERS IN GENOME EDITING (2022)
In vivo base editing rescues Hutchinson-Gilford progeria syndrome in mice
Luke W. Koblan et al.
NATURE (2021)
Gain-of-function cardiomyopathic mutations in RBM20 rewire splicing regulation and re-distribute ribonucleoprotein granules within processing bodies
Aidan M. Fenix et al.
NATURE COMMUNICATIONS (2021)
Twelve years of SAMtools and BCFtools
Petr Danecek et al.
GIGASCIENCE (2021)
In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels
Tanja Rothgangl et al.
NATURE BIOTECHNOLOGY (2021)
Efficient Correction of a Hypertrophic Cardiomyopathy Mutation by ABEmax-NG
Shuhong Ma et al.
CIRCULATION RESEARCH (2021)
Single-molecule, full-length transcript isoform sequencing reveals disease-associated RNA isoforms in cardiomyocytes
Chenchen Zhu et al.
NATURE COMMUNICATIONS (2021)
RBM20-Related Cardiomyopathy: Current Understanding and Future Options
Jan Koelemen et al.
JOURNAL OF CLINICAL MEDICINE (2021)
Genetics of dilated cardiomyopathy: practical implications for heart failure management
Andrew N. Rosenbaum et al.
NATURE REVIEWS CARDIOLOGY (2020)
Continuous evolution of SpCas9 variants compatible with non-G PAMs
Shannon M. Miller et al.
NATURE BIOTECHNOLOGY (2020)
Somatic gene editing ameliorates skeletal and cardiac muscle failure in pig and human models of Duchenne muscular dystrophy
A. Moretti et al.
NATURE MEDICINE (2020)
Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses
Jonathan M. Levy et al.
NATURE BIOMEDICAL ENGINEERING (2020)
Unconstrained genome targeting with near-PAMless engineered CRISPR-Cas9 variants
Russell T. Walton et al.
SCIENCE (2020)
Phage-assisted evolution of an adenine base editor with improved Cas domain compatibility and activity
Michelle F. Richter et al.
NATURE BIOTECHNOLOGY (2020)
Genome editing with CRISPR-Cas nucleases, base editors, transposases and prime editors
Andrew V. Anzalone et al.
NATURE BIOTECHNOLOGY (2020)
AggreCount: an unbiased image analysis tool for identifying and quantifying cellular aggregates in a spatially defined manner
Jacob Aaron Klickstein et al.
JOURNAL OF BIOLOGICAL CHEMISTRY (2020)
Cells of the adult human heart
Monika Litvinukova et al.
NATURE (2020)
GOTI, a method to identify genome-wide off-target effects of genome editing in mouse embryos
Erwei Zuo et al.
NATURE PROTOCOLS (2020)
iPSC Modeling of RBM20-Deficient DCM Identifies Upregulation of RBM20 as a Therapeutic Strategy
Francesca Briganti et al.
CELL REPORTS (2020)
A missense mutation in the RSRSP stretch of Rbm20 causes dilated cardiomyopathy and atrial fibrillation in mice
Kensuke Ihara et al.
SCIENTIFIC REPORTS (2020)
Dysregulated ribonucleoprotein granules promote cardiomyopathy in RBM20 gene-edited pigs
Jay W. Schneider et al.
NATURE MEDICINE (2020)
Identification of a myotropic AAV by massively parallel in vivo evaluation of barcoded capsid variants
Jonas Weinmann et al.
NATURE COMMUNICATIONS (2020)
Protocol for Single-Nucleus Transcriptomics of Diploid and Tetraploid Cardiomyocytes in Murine Hearts
Miao Cui et al.
STAR PROTOCOLS (2020)
CRISPResso2 provides accurate and rapid genome editing sequence analysis
Kendell Clement et al.
NATURE BIOTECHNOLOGY (2019)
The R package Rsubread is easier, faster, cheaper and better for alignment and quantification of RNA sequencing reads
Yang Liao et al.
NUCLEIC ACIDS RESEARCH (2019)
Cytosine base editor generates substantial off-target single-nucleotide variants in mouse embryos
Erwei Zuo et al.
SCIENCE (2019)
Transcriptome-wide off-target RNA editing induced by CRISPR-guided DNA base editors
Julian Grunewald et al.
NATURE (2019)
Circularly permuted and PAM-modified Cas9 variants broaden the targeting scope of base editors
Tony P. Huang et al.
NATURE BIOTECHNOLOGY (2019)
Simple ClinVar: an interactive web server to explore and retrieve gene and disease variants aggregated in ClinVar database
Eduardo Perez-Palma et al.
NUCLEIC ACIDS RESEARCH (2019)
Analysis and minimization of cellular RNA editing by DNA adenine base editors
Holly A. Rees et al.
SCIENCE ADVANCES (2019)
Metascape provides a biologist-oriented resource for the analysis of systems-level datasets
Yingyao Zhou et al.
NATURE COMMUNICATIONS (2019)
Role of Targeted Therapy in Dilated Cardiomyopathy: The Challenging Road Toward a Personalized Approach
Job A. J. Verdonschot et al.
JOURNAL OF THE AMERICAN HEART ASSOCIATION (2019)
Cardiac hypertrophy or failure? - A systematic evaluation of the transverse aortic constriction model in C57BL/6NTac and C57BL/6J substrains
Min Zi et al.
CURRENT RESEARCH IN PHYSIOLOGY (2019)
Strelka2: fast and accurate calling of germline and somatic variants
Sangtae Kim et al.
NATURE METHODS (2018)
In Vivo Ryr2 Editing Corrects Catecholaminergic Polymorphic Ventricular Tachycardia
Xiaolu Pan et al.
CIRCULATION RESEARCH (2018)
Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy
Leonela Amoasii et al.
SCIENCE (2018)
Correction of a pathogenic gene mutation in human embryos
Hong Ma et al.
NATURE (2017)
Recounting Cardiac Cellular Composition
Pingzhu Zhou et al.
CIRCULATION RESEARCH (2016)
A Simplified, Langendorff-Free Method for Concomitant Isolation of Viable Cardiac Myocytes and Nonmyocytes From the Adult Mouse Heart
Matthew Ackers-Johnson et al.
CIRCULATION RESEARCH (2016)
Indel variant analysis of short-read sequencing data with Scalpel
Han Fang et al.
NATURE PROTOCOLS (2016)
Using single nuclei for RNA-seq to capture the transcriptome of postmortem neurons
Suguna Rani Krishnaswami et al.
NATURE PROTOCOLS (2016)
Phospholamban Ablation Using CRISPR/Cas9 System Improves Mortality in a Murine Heart Failure Model
Manami Kaneko et al.
PLOS ONE (2016)
The Rationale and Timing of Molecular Genetic Testing for Dilated Cardiomyopathy
Ana Morales et al.
CANADIAN JOURNAL OF CARDIOLOGY (2015)
Integrating mapping-, assembly- and haplotype-based approaches for calling variants in clinical sequencing applications
Andy Rimmer et al.
NATURE GENETICS (2014)
rMATS: Robust and flexible detection of differential alternative splicing from replicate RNA-Seq data
Shihao Shen et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2014)
Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA
Chengzu Long et al.
SCIENCE (2014)
Moderated estimation of fold change and dispersion for RNA-seq data with DESeq2
Michael I. Love et al.
GENOME BIOLOGY (2014)
STAR: ultrafast universal RNA-seq aligner
Alexander Dobin et al.
BIOINFORMATICS (2013)
REDItools: high-throughput RNA editing detection made easy
Ernesto Picardi et al.
BIOINFORMATICS (2013)
Sensitive detection of somatic point mutations in impure and heterogeneous cancer samples
Kristian Cibulskis et al.
NATURE BIOTECHNOLOGY (2013)
Smart-seq2 for sensitive full-length transcriptome profiling in single cells
Simone Picelli et al.
NATURE METHODS (2013)
Natriuretic Peptides as Predictors of Atrial Fibrillation Recurrences Following Electrical Cardioversion
Theodoros A. Zografos et al.
ARRHYTHMIA & ELECTROPHYSIOLOGY REVIEW (2013)
Genetic variation in the alternative splicing regulator RBM20 is associated with dilated cardiomyopathy
Marwan M. Refaat et al.
HEART RHYTHM (2012)
RBM20, a gene for hereditary cardiomyopathy, regulates titin splicing
Wei Guo et al.
NATURE MEDICINE (2012)
LoFreq: a sequence-quality aware, ultra-sensitive variant caller for uncovering cell-population heterogeneity from high-throughput sequencing datasets
Andreas Wilm et al.
NUCLEIC ACIDS RESEARCH (2012)
Mouse genomic variation and its effect on phenotypes and gene regulation
Thomas M. Keane et al.
NATURE (2011)
A framework for variation discovery and genotyping using next-generation DNA sequencing data
Mark A. DePristo et al.
NATURE GENETICS (2011)
The Genome Analysis Toolkit: A MapReduce framework for analyzing next-generation DNA sequencing data
Aaron McKenna et al.
GENOME RESEARCH (2010)
ANNOVAR: functional annotation of genetic variants from high-throughput sequencing data
Kai Wang et al.
NUCLEIC ACIDS RESEARCH (2010)
Fast and accurate short read alignment with Burrows-Wheeler transform
Heng Li et al.
BIOINFORMATICS (2009)
Fast unfolding of communities in large networks
Vincent D. Blondel et al.
JOURNAL OF STATISTICAL MECHANICS-THEORY AND EXPERIMENT (2008)
Immune responses to adeno-associated virus vectors
AK Zaiss et al.
CURRENT GENE THERAPY (2005)
Vertical agarose gel electrophoresis and electroblotting of high-molecular-weight proteins
CM Warren et al.
ELECTROPHORESIS (2003)
dbSNP: the NCBI database of genetic variation
ST Sherry et al.
NUCLEIC ACIDS RESEARCH (2001)