CRISPR/Cas Applications in Myotonic Dystrophy: Expanding Opportunities
Published 2019 View Full Article
- Home
- Publications
- Publication Search
- Publication Details
Title
CRISPR/Cas Applications in Myotonic Dystrophy: Expanding Opportunities
Authors
Keywords
-
Journal
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
Volume 20, Issue 15, Pages 3689
Publisher
MDPI AG
Online
2019-07-29
DOI
10.3390/ijms20153689
References
Ask authors/readers for more resources
Related references
Note: Only part of the references are listed.- Adeno-associated virus vector as a platform for gene therapy delivery
- (2019) Dan Wang et al. NATURE REVIEWS DRUG DISCOVERY
- Identification of preexisting adaptive immunity to Cas9 proteins in humans
- (2019) Carsten T. Charlesworth et al. NATURE MEDICINE
- Adopt a moratorium on heritable genome editing
- (2019) Eric S. Lander et al. NATURE
- Repeat-associated non-AUG (RAN) translation: insights from pathology
- (2019) Monica Banez-Coronel et al. LABORATORY INVESTIGATION
- Manage risk of accidental gene editing of germline
- (2019) Darren G. Monckton NATURE
- Evaluation and Reduction of CRISPR Off-Target Cleavage Events
- (2019) Christopher A. Vakulskas et al. Nucleic Acid Therapeutics
- CRISPR base editors: genome editing without double-stranded breaks
- (2018) Ayman Eid et al. BIOCHEMICAL JOURNAL
- Rescue of Fragile X Syndrome Neurons by DNA Methylation Editing of the FMR1 Gene
- (2018) X. Shawn Liu et al. CELL
- Trinucleotide repeat instability during double-strand break repair: from mechanisms to gene therapy
- (2018) Valentine Mosbach et al. CURRENT GENETICS
- De novo repeat interruptions are associated with reduced somatic instability and mild or absent clinical features in myotonic dystrophy type 1
- (2018) Sarah A. Cumming et al. EUROPEAN JOURNAL OF HUMAN GENETICS
- A CRISPR–dCas Toolbox for Genetic Engineering and Synthetic Biology
- (2018) Xiaoshu Xu et al. JOURNAL OF MOLECULAR BIOLOGY
- Efficient CRISPR/Cas9-mediated editing of trinucleotide repeat expansion in myotonic dystrophy patient-derived iPS and myogenic cells
- (2018) Sumitava Dastidar et al. NUCLEIC ACIDS RESEARCH
- CRISPR/Cas9-Targeted Deletion of Polyglutamine in Spinocerebellar Ataxia Type 3-Derived Induced Pluripotent Stem Cells
- (2018) Shuming Ouyang et al. STEM CELLS AND DEVELOPMENT
- TALEN-Induced Double-Strand Break Repair of CTG Trinucleotide Repeats
- (2018) Valentine Mosbach et al. Cell Reports
- RNA-dependent RNA targeting by CRISPR-Cas9
- (2018) Steven C Strutt et al. eLife
- Precise Excision of the CAG Tract from the Huntingtin Gene by Cas9 Nickases
- (2018) Magdalena Dabrowska et al. Frontiers in Neuroscience
- Small Molecules Which Improve Pathogenesis of Myotonic Dystrophy Type 1
- (2018) Marta López-Morató et al. Frontiers in Neurology
- Abnormalities in Skeletal Muscle Myogenesis, Growth, and Regeneration in Myotonic Dystrophy
- (2018) Laurène M. André et al. Frontiers in Neurology
- RNA-mediated therapies in myotonic dystrophy
- (2018) Sarah J. Overby et al. DRUG DISCOVERY TODAY
- Gene and cell and therapy for muscular dystrophies: are we getting there?
- (2018) Francesco Galli et al. HUMAN GENE THERAPY
- Therapeutic Genome Editing for Myotonic Dystrophy Type 1 Using CRISPR/Cas9
- (2018) Yanlin Wang et al. MOLECULAR THERAPY
- CRISPR-Cas guides the future of genetic engineering
- (2018) Gavin J. Knott et al. SCIENCE
- Targeted Reactivation of FMR1 Transcription in Fragile X Syndrome Embryonic Stem Cells
- (2018) Jill M. Haenfler et al. Frontiers in Molecular Neuroscience
- Unraveling the Complex Story of Immune Responses to AAV Vectors Trial After Trial
- (2017) Céline Vandamme et al. HUMAN GENE THERAPY
- CRISPR/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of Huntington’s disease
- (2017) Su Yang et al. JOURNAL OF CLINICAL INVESTIGATION
- Impeding Transcription of Expanded Microsatellite Repeats by Deactivated Cas9
- (2017) Belinda S. Pinto et al. MOLECULAR CELL
- CRISPR/Cas9-Induced (CTG⋅CAG) n Repeat Instability in the Myotonic Dystrophy Type 1 Locus: Implications for Therapeutic Genome Editing
- (2017) Ellen L. van Agtmaal et al. MOLECULAR THERAPY
- CRISPR/Cas9 Editing of the Mutant Huntingtin Allele In Vitro and In Vivo
- (2017) Alex Mas Monteys et al. MOLECULAR THERAPY
- Antisense transcription of the myotonic dystrophy locus yields low-abundant RNAs with and without (CAG)n repeat
- (2017) Anke E. E. G. Gudde et al. RNA Biology
- CRISPR/Cas9-Mediated Deletion of CTG Expansions Recovers Normal Phenotype in Myogenic Cells Derived from Myotonic Dystrophy 1 Patients
- (2017) Claudia Provenzano et al. Molecular Therapy-Nucleic Acids
- Reversal of Phenotypic Abnormalities by CRISPR/Cas9-Mediated Gene Correction in Huntington Disease Patient-Derived Induced Pluripotent Stem Cells
- (2017) Xiaohong Xu et al. Stem Cell Reports
- Deletion of the GAA repeats from the human frataxin gene using the CRISPR-Cas9 system in YG8R-derived cells and mouse models of Friedreich ataxia
- (2016) D L Ouellet et al. GENE THERAPY
- Dmpkgene deletion or antisense knockdown does not compromise cardiac or skeletal muscle function in mice
- (2016) Samuel T. Carrell et al. HUMAN MOLECULAR GENETICS
- Permanent inactivation of Huntington's disease mutation by personalized allele-specific CRISPR/Cas9
- (2016) Jun Wan Shin et al. HUMAN MOLECULAR GENETICS
- Genome Therapy of Myotonic Dystrophy Type 1 iPS Cells for Development of Autologous Stem Cell Therapy
- (2016) Yuanzheng Gao et al. MOLECULAR THERAPY
- Unravelling the myotonic dystrophy type 1 clinical spectrum: A systematic registry-based study with implications for disease classification
- (2016) M. De Antonio et al. REVUE NEUROLOGIQUE
- Generation of an isogenic, gene-corrected control cell line of the spinocerebellar ataxia type 2 patient-derived iPSC line H271
- (2016) Adele G. Marthaler et al. Stem Cell Research
- Contracting CAG/CTG repeats using the CRISPR-Cas9 nickase
- (2016) Cinzia Cinesi et al. Nature Communications
- Reactivation of FMR1 by CRISPR/Cas9-Mediated Deletion of the Expanded CGG-Repeat of the Fragile X Chromosome
- (2016) Nina Xie et al. PLoS One
- Pericytes in the myovascular niche promote post-natal myofiber growth and satellite cell quiescence
- (2015) E. Kostallari et al. DEVELOPMENT
- Perspective on Adeno-Associated Virus Capsid Modification for Duchenne Muscular Dystrophy Gene Therapy
- (2015) Michael E. Nance et al. HUMAN GENE THERAPY
- Expanding the Biologist’s Toolkit with CRISPR-Cas9
- (2015) Samuel H. Sternberg et al. MOLECULAR CELL
- Beyond editing: repurposing CRISPR–Cas9 for precision genome regulation and interrogation
- (2015) Antonia A. Dominguez et al. NATURE REVIEWS MOLECULAR CELL BIOLOGY
- Pericytes are Essential for Skeletal Muscle Formation
- (2015) Alexander Birbrair et al. Stem Cell Reviews and Reports
- Genome Modification Leads to Phenotype Reversal in Human Myotonic Dystrophy Type 1 Induced Pluripotent Stem Cell-Derived Neural Stem Cells
- (2015) Guangbin Xia et al. STEM CELLS
- Reversion of FMR1 Methylation and Silencing by Editing the Triplet Repeats in Fragile X iPSC-Derived Neurons
- (2015) Chul-Yong Park et al. Cell Reports
- Pericytes are Essential for Skeletal Muscle Formation
- (2015) Alexander Birbrair et al. Stem Cell Reviews and Reports
- Efficient genome modification by CRISPR-Cas9 nickase with minimal off-target effects
- (2014) Bin Shen et al. NATURE METHODS
- Highly Specific Contractions of a Single CAG/CTG Trinucleotide Repeat by TALEN in Yeast
- (2014) Guy-Franck Richard et al. PLoS One
- RNA toxicity in human disease and animal models: From the uncovering of a new mechanism to the development of promising therapies
- (2013) Géraldine Sicot et al. BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE
- Double Nicking by RNA-Guided CRISPR Cas9 for Enhanced Genome Editing Specificity
- (2013) F. Ann Ran et al. CELL
- Repair of Strand Breaks by Homologous Recombination
- (2013) M. Jasin et al. Cold Spring Harbor Perspectives in Biology
- Detection of Slipped-DNAs at the Trinucleotide Repeats of the Myotonic Dystrophy Type I Disease Locus in Patient Tissues
- (2013) Michelle M. Axford et al. PLoS Genetics
- Gene transfer to skeletal muscle by site-specific delivery of electroporation and ultrasound
- (2012) Yuanli Li et al. BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS
- Markedly enhanced skeletal muscle transfection achieved by the ultrasound-targeted delivery of non-viral gene nanocarriers with microbubbles
- (2012) Caitlin W. Burke et al. JOURNAL OF CONTROLLED RELEASE
- Adeno-Associated Virus Antibody Profiles in Newborns, Children, and Adolescents
- (2011) Roberto Calcedo et al. Clinical and Vaccine Immunology
- Pericytes resident in postnatal skeletal muscle differentiate into muscle fibres and generate satellite cells
- (2011) A. Dellavalle et al. Nature Communications
- Regulation of Homologous Recombination in Eukaryotes
- (2010) Wolf-Dietrich Heyer et al. Annual Review of Genetics
- Variant CCG and GGC repeats within the CTG expansion dramatically modify mutational dynamics and likely contribute toward unusual symptoms in some myotonic dystrophy type 1 patients
- (2010) Claudia Braida et al. HUMAN MOLECULAR GENETICS
- Replication-dependent instability at (CTG)•(CAG) repeat hairpins in human cells
- (2010) Guoqi Liu et al. Nature Chemical Biology
- Non-ATG-initiated translation directed by microsatellite expansions
- (2010) T. Zu et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- A muscle-targeting peptide displayed on AAV2 improves muscle tropism on systemic delivery
- (2009) C-Y Yu et al. GENE THERAPY
- Triplet-repeat oligonucleotide-mediated reversal of RNA toxicity in myotonic dystrophy
- (2009) S. A. M. Mulders et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Zinc-finger directed double-strand breaks within CAG repeat tracts promote repeat instability in human cells
- (2009) D. Mittelman et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Reversal of RNA Dominance by Displacement of Protein Sequestered on Triplet Repeat RNA
- (2009) T. M. Wheeler et al. SCIENCE
Find Funding. Review Successful Grants.
Explore over 25,000 new funding opportunities and over 6,000,000 successful grants.
ExploreDiscover Peeref hubs
Discuss science. Find collaborators. Network.
Join a conversation