Related references
Note: Only part of the references are listed.The sustained expression of Cas9 targeting toxic RNAs reverses disease phenotypes in mouse models of myotonic dystrophy type 1
Ranjan Batra et al.
NATURE BIOMEDICAL ENGINEERING (2021)
Myotonic dystrophy type 1 drug development: A pipeline toward the market
Marta Pascual-Gilabert et al.
DRUG DISCOVERY TODAY (2021)
Paving the way towards precise and safe CRISPR genome editing
Pawel Sledzinski et al.
BIOTECHNOLOGY ADVANCES (2021)
Design of novel small molecule base-pair recognizers of toxic CUG RNA transcripts characteristics of DM1
Raul Ondono et al.
COMPUTATIONAL AND STRUCTURAL BIOTECHNOLOGY JOURNAL (2021)
Unexpected Mutations by CRISPR-Cas9 CTG Repeat Excision in Myotonic Dystrophy and Use of CRISPR Interference as an Alternative Approach
Miki Ikeda et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2020)
Cas9 Cuts and Consequences; Detecting, Predicting, and Mitigating CRISPR/Cas9 On- and Off-Target Damage Techniques for Detecting, Predicting, and Mitigating the On- and off-target Effects of Cas9 Editing
Anthony Newman et al.
BIOESSAYS (2020)
Resection and repair of a Cas9 double-strand break at CTG trinucleotide repeats induces local and extensive chromosomal deletions
Valentine Mosbach et al.
PLOS GENETICS (2020)
INDEL detection, the 'Achilles heel' of precise genome editing: a survey of methods for accurate profiling of gene editing induced indels
Eric Paul Bennett et al.
NUCLEIC ACIDS RESEARCH (2020)
CRISPResso2 provides accurate and rapid genome editing sequence analysis
Kendell Clement et al.
NATURE BIOTECHNOLOGY (2019)
Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy
Christopher E. Nelson et al.
NATURE MEDICINE (2019)
CRISPR-Cas9 genome editing induces megabase-scale chromosomal truncations
Gregoire Cullot et al.
NATURE COMMUNICATIONS (2019)
Collateral damage and CRISPR genome editing
Mark Thomas et al.
PLOS GENETICS (2019)
CRISPR/Cas Applications in Myotonic Dystrophy: Expanding Opportunities
Renee H. L. Raaijmakers et al.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2019)
Genome Editing of Expanded CTG Repeats within the Human DMPK Gene Reduces Nuclear RNA Foci in the Muscle of DM1 Mice
Mirella Lo Scrudato et al.
MOLECULAR THERAPY (2019)
Recovery in the Myogenic Program of Congenital Myotonic Dystrophy Myoblasts after Excision of the Expanded (CTG)n Repeat
Laurene M. Andre et al.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2019)
Target-Specific Precision of CRISPR-Mediated Genome Editing
Anob M. Chakrabarti et al.
MOLECULAR CELL (2019)
Predicting the mutations generated by repair of Cas9-induced double-strand breaks
Felicity Allen et al.
NATURE BIOTECHNOLOGY (2019)
AmpUMI: design and analysis of unique molecular identifiers for deep amplicon sequencing
Kendell Clement et al.
BIOINFORMATICS (2018)
Repair of double-strand breaks induced by CRISPR-Cas9 leads to large deletions and complex rearrangements
Michael Kosicki et al.
NATURE BIOTECHNOLOGY (2018)
Efficient CRISPR/Cas9-mediated editing of trinucleotide repeat expansion in myotonic dystrophy patient-derived iPS and myogenic cells
Sumitava Dastidar et al.
NUCLEIC ACIDS RESEARCH (2018)
No unexpected CRISPR-Cas9 off-target activity revealed by trio sequencing of gene-edited mice
Vivek Iyer et al.
PLOS GENETICS (2018)
Abnormalities in Skeletal Muscle Myogenesis, Growth, and Regeneration in Myotonic Dystrophy
Laurene M. Andre et al.
FRONTIERS IN NEUROLOGY (2018)
Therapeutic Genome Editing for Myotonic Dystrophy Type 1 Using CRISPR/Cas9
Yanlin Wang et al.
MOLECULAR THERAPY (2018)
Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing Therapeutics
Brittany E. Givens et al.
AAPS JOURNAL (2018)
CRISPR-Cas9 off-targeting assessment with nucleic acid duplex energy parameters
Ferhat Alkan et al.
GENOME BIOLOGY (2018)
Myotonic Dystrophy and Developmental Regulation of RNA Processing
James D. Thomas et al.
COMPREHENSIVE PHYSIOLOGY (2018)
Impeding Transcription of Expanded Microsatellite Repeats by Deactivated Cas9
Belinda S. Pinto et al.
MOLECULAR CELL (2017)
CRISPR/Cas9-Induced (CTG.GAG)n, Repeat Instability in the Myotonic Dystrophy Type 1 Locus: Implications for Therapeutic Genome Editing
Ellen L. van Agtmaal et al.
MOLECULAR THERAPY (2017)
Non-homologous DNA end joining and alternative pathways to double-strand break repair
Howard H. Y. Chang et al.
NATURE REVIEWS MOLECULAR CELL BIOLOGY (2017)
Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy
Niclas E. Bengtsson et al.
NATURE COMMUNICATIONS (2017)
Downregulation of the Glial GLT1 Glutamate Transporter and Purkinje Cell Dysfunction in a Mouse Model of Myotonic Dystrophy
Geraldine Sicot et al.
CELL REPORTS (2017)
A machine learning approach for predicting CRISPR-Cas9 cleavage efficiencies and patterns underlying its mechanism of action
Shiran Abadi et al.
PLOS COMPUTATIONAL BIOLOGY (2017)
Targeting DMPK with Antisense Oligonucleotide Improves Muscle Strength in Myotonic Dystrophy Type 1 Mice
Dominic Jauvin et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2017)
CRISPR/Cas9-Mediated Deletion of CTG Expansions Recovers Normal Phenotype in Myogenic Cells Derived from Myotonic Dystrophy 1 Patients
Claudia Provenzano et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2017)
Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA in vivo induces homology-directed DNA repair
Kunwoo Lee et al.
NATURE BIOMEDICAL ENGINEERING (2017)
Myotonic dystrophy: candidate small molecule therapeutics
Piotr Konieczny et al.
DRUG DISCOVERY TODAY (2017)
Elimination of Toxic Microsatellite Repeat Expansion RNA by RNA-Targeting Cas9
Ranjan Batra et al.
CELL (2017)
Rationally engineered Cas9 nucleases with improved specificity
Ian M. Slaymaker et al.
SCIENCE (2016)
Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy
Chengzu Long et al.
SCIENCE (2016)
In vivo gene editing in dystrophic mouse muscle and muscle stem cells
Mohammadsharif Tabebordbar et al.
SCIENCE (2016)
In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy
Christopher E. Nelson et al.
SCIENCE (2016)
The democratization of gene editing: Insights from site-specific cleavage and double-strand break repair
Maria Jasin et al.
DNA REPAIR (2016)
Reliable and versatile immortal muscle cell models from healthy and myotonic dystrophy type 1 primary human myoblasts
Boris Pantic et al.
EXPERIMENTAL CELL RESEARCH (2016)
MicroRNA-222 regulates muscle alternative splicing through Rbm24 during differentiation of skeletal muscle cells
B. Cardinali et al.
CELL DEATH & DISEASE (2016)
Myotonic dystrophies: An update on clinical aspects, genetic, pathology, and molecular pathomechanisms
Giovanni Meola et al.
BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE (2015)
Therapeutic impact of systemic AAV-mediated RNA interference in a mouse model of myotonic dystrophy
Darren R. Bisset et al.
HUMAN MOLECULAR GENETICS (2015)
Short antisense-locked nucleic acids (all-LNAs) correct alternative splicing abnormalities in myotonic dystrophy
Agnieszka Wojtkowiak-Szlachcic et al.
NUCLEIC ACIDS RESEARCH (2015)
Off-target Effects in CRISPR/Cas9-mediated Genome Engineering
Xiao-Hui Zhang et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2015)
Biomolecular identification of (CCTG)n mutation in myotonic dystrophy type 2 (DM2) by FISH on muscle biopsy
R Cardani et al.
EUROPEAN JOURNAL OF HISTOCHEMISTRY (2015)
Cas-OFFinder: a fast and versatile algorithm that searches for potential off-target sites of Cas9 RNA-guided endonucleases
Sangsu Bae et al.
BIOINFORMATICS (2014)
Programmable RNA recognition and cleavage by CRISPR/Cas9
Mitchell R. O'Connell et al.
NATURE (2014)
MBNL proteins and their target RNAs, interaction and splicing regulation
Patryk Konieczny et al.
NUCLEIC ACIDS RESEARCH (2014)
Structures of Cas9 Endonucleases Reveal RNA-Mediated Conformational Activation
Martin Jinek et al.
SCIENCE (2014)
Reducing INDEL calling errors in whole genome and exome sequencing data
Han Fang et al.
GENOME MEDICINE (2014)
Noncoding RNAs: Emerging Players in Muscular Dystrophies
Germana Falcone et al.
BIOMED RESEARCH INTERNATIONAL (2014)
COSMID: A Web-based Tool for Identifying and Validating CRISPR/Cas Off-target Sites
Thomas J. Cradick et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2014)
Splicing Biomarkers of Disease Severity in Myotonic Dystrophy
Masayuki Nakamori et al.
ANNALS OF NEUROLOGY (2013)
RNA toxicity in human disease and animal models: From the uncovering of a new mechanism to the development of promising therapies
Geraldine Sicot et al.
BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE (2013)
Myotonic dystrophy CTG expansion affects synaptic vesicle proteins, neurotransmission and mouse behaviour
Oscar Hernandez-Hernandez et al.
BRAIN (2013)
RNA-Guided Human Genome Engineering via Cas9
Prashant Mali et al.
SCIENCE (2013)
Multiplex Genome Engineering Using CRISPR/Cas Systems
Le Cong et al.
SCIENCE (2013)
Targeting nuclear RNA for in vivo correction of myotonic dystrophy
Thurman M. Wheeler et al.
NATURE (2012)
RNA-guided genetic silencing systems in bacteria and archaea
Blake Wiedenheft et al.
NATURE (2012)
A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity
Martin Jinek et al.
SCIENCE (2012)
Molecular, Physiological, and Motor Performance Defects in DMSXL Mice Carrying >1,000 CTG Repeats from the Human DM1 Locus
Aline Huguet et al.
PLOS GENETICS (2012)
FLASH: fast length adjustment of short reads to improve genome assemblies
Tanja Magoc et al.
BIOINFORMATICS (2011)
Dysregulation and cellular mislocalization of specific miRNAs in myotonic dystrophy type 1
Riccardo Perbellini et al.
NEUROMUSCULAR DISORDERS (2011)
Non-ATG-initiated translation directed by microsatellite expansions
Tao Zu et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2011)
Myotonic dystrophy mouse models: towards rational therapy development
Mario Gomes-Pereira et al.
TRENDS IN MOLECULAR MEDICINE (2011)
The Genome Analysis Toolkit: A MapReduce framework for analyzing next-generation DNA sequencing data
Aaron McKenna et al.
GENOME RESEARCH (2010)
Elevation of RNA-binding protein CUGBP1 is an early event in an inducible heart-specific mouse model of myotonic dystrophy
Guey-Shin Wang et al.
JOURNAL OF CLINICAL INVESTIGATION (2007)
CTG trinucleotide repeat big jumps: Large expansions, small mice
Mario Gomes-Pereira et al.
PLOS GENETICS (2007)
CRISPR provides acquired resistance against viruses in prokaryotes
Rodolphe Barrangou et al.
SCIENCE (2007)
Myotonic dystrophy type 1 is associated with nuclear foci of mutant RNA, sequestration of muscleblind proteins and deregulated alternative splicing in neurons
H Jiang et al.
HUMAN MOLECULAR GENETICS (2004)
Myotonic dystrophy in transgenic mice expressing an expanded CUG repeat
A Mankodi et al.
SCIENCE (2000)
Transgenic mice carrying large human genomic sequences with expanded CTG repeat mimic closely the DM CTG repeat intergenerational and somatic instability
H Seznec et al.
HUMAN MOLECULAR GENETICS (2000)