Therapeutic Strategies for Dystrophin Replacement in Duchenne Muscular Dystrophy
Published 2022 View Full Article
- Home
- Publications
- Publication Search
- Publication Details
Title
Therapeutic Strategies for Dystrophin Replacement in Duchenne Muscular Dystrophy
Authors
Keywords
-
Journal
Frontiers in Medicine
Volume 9, Issue -, Pages -
Publisher
Frontiers Media SA
Online
2022-03-30
DOI
10.3389/fmed.2022.859930
References
Ask authors/readers for more resources
Related references
Note: Only part of the references are listed.- Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMDmdx rat model
- (2022) Audrey Bourdon et al. GENE THERAPY
- After Patient Death, FDA Places Hold on Pfizer Duchenne Muscular Dystrophy Gene Therapy Trial
- (2022) Alex Philippidis HUMAN GENE THERAPY
- CRISPR-Cas9 Gene Therapy for Duchenne Muscular Dystrophy
- (2022) Cedric Happi Mbakam et al. Neurotherapeutics
- CRISPR/Cas9 editing of directly reprogrammed myogenic progenitors restores dystrophin expression in a mouse model of muscular dystrophy
- (2022) Seraina A. Domenig et al. Stem Cell Reports
- Insertion of the Icelandic Mutation (A673T) by Prime Editing: A Potential Preventive Treatment for Familial and Sporadic Alzheimer's Disease
- (2022) Guillaume Tremblay et al. CRISPR Journal
- Lack of toxicity in non-human primates receiving clinically relevant doses of an AAV9.U7snRNA vector designed to induce DMD exon 2 skipping
- (2021) Liubov V Gushchina et al. HUMAN GENE THERAPY
- Assessment of rAAVrh.74.MHCK7.micro-dystrophin Gene Therapy Using Magnetic Resonance Imaging in Children With Duchenne Muscular Dystrophy
- (2021) Rebecca J. Willcocks et al. JAMA Network Open
- Micro-dystrophin gene therapy prevents heart failure in an improved Duchenne muscular dystrophy cardiomyopathy mouse model
- (2021) Zachary M. Howard et al. JCI Insight
- Proteomic analysis identifies key differences in the cardiac interactomes of dystrophin and micro-dystrophin
- (2021) Hong Wang et al. HUMAN MOLECULAR GENETICS
- Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice
- (2021) Pengpeng Liu et al. Nature Communications
- Precise correction of Duchenne muscular dystrophy exon deletion mutations by base and prime editing
- (2021) F. Chemello et al. Science Advances
- Absence of Significant Off-Target Splicing Variation with a U7snRNA Vector Targeting DMD Exon 2 Duplications
- (2021) Nicolas Wein et al. HUMAN GENE THERAPY
- AAV integration in human hepatocytes
- (2021) Dhwanil A. Dalwadi et al. MOLECULAR THERAPY
- Efficient precise in vivo base editing in adult dystrophic mice
- (2021) Li Xu et al. Nature Communications
- Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping
- (2021) Tabatha R. Simmons et al. Molecular Therapy-Methods & Clinical Development
- Enhanced prime editing systems by manipulating cellular determinants of editing outcomes
- (2021) Peter J. Chen et al. CELL
- Fourth Boy Dies in Clinical Trial of Astellas' AT132
- (2021) Alex Philippidis HUMAN GENE THERAPY
- The Effect of Immunomodulatory Treatments on Anti-Dystrophin Immune Response After AAV Gene Therapy in Dystrophin Deficient mdx Mice
- (2021) Ning Li et al. Journal of Neuromuscular Diseases
- Engineering adeno-associated virus vectors for gene therapy
- (2020) Chengwen Li et al. NATURE REVIEWS GENETICS
- Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy
- (2020) Diane E. Frank et al. NEUROLOGY
- Correction: Hybrid Adeno-Associated Viral Vectors Utilizing Transposase-Mediated Somatic Integration for Stable Transgene Expression in Human Cells
- (2020) Wenli Zhang et al. PLoS One
- Enhanced CRISPR-Cas9 correction of Duchenne muscular dystrophy in mice by a self-complementary AAV delivery system
- (2020) Yu Zhang et al. Science Advances
- Unconstrained genome targeting with near-PAMless engineered CRISPR-Cas9 variants
- (2020) Russell T. Walton et al. SCIENCE
- Identification of therapeutics that target eEF1A2 and upregulate utrophin A translation in dystrophic muscles
- (2020) Christine Péladeau et al. Nature Communications
- Extracellular nanovesicles for packaging of CRISPR-Cas9 protein and sgRNA to induce therapeutic exon skipping
- (2020) Peter Gee et al. Nature Communications
- Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping
- (2020) Paula R. Clemens et al. JAMA Neurology
- Global epidemiology of Duchenne muscular dystrophy: an updated systematic review and meta-analysis
- (2020) Salvatore Crisafulli et al. Orphanet Journal of Rare Diseases
- Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy
- (2020) Jerry R. Mendell et al. JAMA Neurology
- Therapeutic Strategies for Duchenne Muscular Dystrophy: An Update
- (2020) Chengmei Sun et al. Genes
- After Third Death, Audentes' AT132 Remains on Clinical Hold
- (2020) Alex Philippidis HUMAN GENE THERAPY
- Base editing: advances and therapeutic opportunities
- (2020) Elizabeth M. Porto et al. NATURE REVIEWS DRUG DISCOVERY
- Genome Editing-Mediated Utrophin Upregulation in Duchenne Muscular Dystrophy Stem Cells
- (2020) Kasturi Sengupta et al. Molecular Therapy-Nucleic Acids
- Viltolarsen in Japanese Duchenne muscular dystrophy patients: A phase 1/2 study
- (2020) Hirofumi Komaki et al. Annals of Clinical and Translational Neurology
- Viral Delivery Systems for CRISPR
- (2019) Christine Xu et al. Viruses-Basel
- CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells
- (2019) Yi-Li Min et al. Science Advances
- Cytosine base editor generates substantial off-target single-nucleotide variants in mouse embryos
- (2019) Erwei Zuo et al. SCIENCE
- Genome editing: A perspective on the application of CRISPR/Cas9 to study human diseases (Review)
- (2019) Diana Rodr�guez‑Rodr�guez et al. INTERNATIONAL JOURNAL OF MOLECULAR MEDICINE
- Intein-mediated protein trans-splicing expands adeno-associated virus transfer capacity in the retina
- (2019) Patrizia Tornabene et al. Science Translational Medicine
- Antisense Oligonucleotide Therapies for Neurodegenerative Diseases
- (2019) C. Frank Bennett et al. Annual Review of Neuroscience
- Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53
- (2019) Claudia Brogna et al. PLoS One
- Skeletal muscle cell transplantation: models and methods
- (2019) Amber L. Mueller et al. JOURNAL OF MUSCLE RESEARCH AND CELL MOTILITY
- Dual-AAV delivery of large gene sequences to the inner ear
- (2019) Ellen Reisinger HEARING RESEARCH
- How the central domain of dystrophin acts to bridge F-actin to sarcolemmal lipids
- (2019) Dominique Mias-Lucquin et al. JOURNAL OF STRUCTURAL BIOLOGY
- Search-and-replace genome editing without double-strand breaks or donor DNA
- (2019) Andrew V. Anzalone et al. NATURE
- P.140RNA-Seq shows an absence of off-target splicing effects in AAV9-U7snRNA mediated skipping of DMD exon 2
- (2019) K. Flanigan et al. NEUROMUSCULAR DISORDERS
- Stem cell-based therapies for Duchenne muscular dystrophy
- (2019) Congshan Sun et al. EXPERIMENTAL NEUROLOGY
- Restoration of Functional Full-Length Dystrophin After Intramuscular Transplantation of Foamy Virus-Transduced Myoblasts
- (2019) Jinhong Meng et al. HUMAN GENE THERAPY
- Utrophin up-regulation by artificial transcription factors induces muscle rescue and impacts the neuromuscular junction in mdx mice
- (2018) Cinzia Pisani et al. BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE
- Dynamics of Dystrophin’s Actin-Binding Domain
- (2018) Michael E. Fealey et al. BIOPHYSICAL JOURNAL
- Advances in therapeutic use of a drug-stimulated translational readthrough of premature termination codons
- (2018) Maciej Dabrowski et al. MOLECULAR MEDICINE
- Systemic AAV micro-dystrophin gene therapy for Duchenne muscular dystrophy
- (2018) Dongsheng Duan MOLECULAR THERAPY
- Functional Rescue of Dystrophin Deficiency in Mice Caused by Frameshift Mutations Using Campylobacter jejuni Cas9
- (2018) Taeyoung Koo et al. MOLECULAR THERAPY
- CRISPR-Induced Deletion with SaCas9 Restores Dystrophin Expression in Dystrophic Models In Vitro and In Vivo
- (2018) Benjamin L. Duchêne et al. MOLECULAR THERAPY
- Base editing: precision chemistry on the genome and transcriptome of living cells
- (2018) Holly A. Rees et al. NATURE REVIEWS GENETICS
- HLA Polymorphism Affects Risk of de novo Mutation of dystrophin Gene and Clinical Severity of Duchenne Muscular Dystrophy in a Southern Chinese Population
- (2018) Huan Li et al. Frontiers in Neurology
- S/MAR Element Facilitates Episomal Long-Term Persistence of Adeno-Associated Virus Vector Genomes in Proliferating Cells
- (2017) Claudia Hagedorn et al. HUMAN GENE THERAPY
- Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
- (2017) Craig M McDonald et al. LANCET
- Antisense Oligonucleotide-Based Therapy for Neuromuscular Disease
- (2017) Valentina Sardone et al. MOLECULES
- PhaseOut DMD: a Phase 2, proof of concept, clinical study of utrophin modulation with ezutromid
- (2017) F. Muntoni et al. NEUROMUSCULAR DISORDERS
- The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review
- (2017) S. Ryder et al. Orphanet Journal of Rare Diseases
- Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy
- (2017) Caroline Le Guiner et al. Nature Communications
- FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga
- (2017) Annemieke Aartsma-Rus et al. Nucleic Acid Therapeutics
- Cell Therapy in Myology: Dynamics of Muscle Precursor Cell Death after Intramuscular Administration in Non-human Primates
- (2017) Daniel Skuk et al. Molecular Therapy-Methods & Clinical Development
- A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model of Duchenne Muscular Dystrophy
- (2017) Chady H. Hakim et al. Molecular Therapy-Methods & Clinical Development
- Effective Depletion of Pre-existing Anti-AAV Antibodies Requires Broad Immune Targeting
- (2017) Victoria M. Velazquez et al. Molecular Therapy-Methods & Clinical Development
- CRISPR/Cas9 in Genome Editing and Beyond
- (2016) Haifeng Wang et al. Annual Review of Biochemistry
- CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice
- (2016) Li Xu et al. MOLECULAR THERAPY
- Histological effects of givinostat in boys with Duchenne muscular dystrophy
- (2016) Paolo Bettica et al. NEUROMUSCULAR DISORDERS
- GeneBase 1.1: a tool to summarize data from NCBI gene datasets and its application to an update of human gene statistics
- (2016) Allison Piovesan et al. Database-The Journal of Biological Databases and Curation
- In vivo tissue-tropism of adeno-associated viral vectors
- (2016) Arun Srivastava Current Opinion in Virology
- Efficient Restoration of the Dystrophin Gene Reading Frame and Protein Structure in DMD Myoblasts Using the CinDel Method
- (2016) Jean-Paul Iyombe-Engembe et al. Molecular Therapy-Nucleic Acids
- Dystrophin Genotype–Cardiac Phenotype Correlations in Duchenne and Becker Muscular Dystrophies Using Cardiac Magnetic Resonance Imaging
- (2015) Animesh Tandon et al. AMERICAN JOURNAL OF CARDIOLOGY
- Advances in genetic therapeutic strategies for Duchenne muscular dystrophy
- (2015) Simon Guiraud et al. EXPERIMENTAL PHYSIOLOGY
- The TREAT-NMD DMD Global Database: Analysis of More than 7,000 Duchenne Muscular Dystrophy Mutations
- (2015) Catherine L. Bladen et al. HUMAN MUTATION
- The first exon duplication mouse model of Duchenne muscular dystrophy: A tool for therapeutic development
- (2015) Adeline Vulin et al. NEUROMUSCULAR DISORDERS
- First Study of Intra-Arterial Delivery of Myogenic Mononuclear Cells to Skeletal Muscles in Primates
- (2014) Daniel Skuk et al. CELL TRANSPLANTATION
- HDAC-regulated myomiRs control BAF60 variant exchange and direct the functional phenotype of fibro-adipogenic progenitors in dystrophic muscles
- (2014) V. Saccone et al. GENES & DEVELOPMENT
- EMA reconsiders 'read-through' drug against Duchenne muscular dystrophy following appeal
- (2014) Asher Mullard NATURE BIOTECHNOLOGY
- Histone deacetylase inhibitors: a potential epigenetic treatment for Duchenne muscular dystrophy
- (2014) Silvia Consalvi et al. Epigenomics
- Stem Cell Transplantation for Muscular Dystrophy: The Challenge of Immune Response
- (2014) Sara Martina Maffioletti et al. Biomed Research International
- Tissue-resident mesenchymal stem/progenitor cells in skeletal muscle: collaborators or saboteurs?
- (2013) Robert N. Judson et al. FEBS Journal
- Triple Trans-Splicing Adeno-Associated Virus Vectors Capable of Transferring the Coding Sequence for Full-Length Dystrophin Protein into Dystrophic Mice
- (2013) Taeyoung Koo et al. HUMAN GENE THERAPY
- Plasmapheresis Eliminates the Negative Impact of AAV Antibodies on Microdystrophin Gene Expression Following Vascular Delivery
- (2013) LG Chicoine et al. MOLECULAR THERAPY
- Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches
- (2013) Rebecca J. Fairclough et al. NATURE REVIEWS GENETICS
- Satellite Cells and the Muscle Stem Cell Niche
- (2013) Hang Yin et al. PHYSIOLOGICAL REVIEWS
- Exon 45 Skipping Through U1-snRNA Antisense Molecules Recovers the Dys-nNOS Pathway and Muscle Differentiation in Human DMD Myoblasts
- (2012) Valentina Cazzella et al. MOLECULAR THERAPY
- Engineering Multiple U7snRNA Constructs to Induce Single and Multiexon-skipping for Duchenne Muscular Dystrophy
- (2012) Aurélie Goyenvalle et al. MOLECULAR THERAPY
- Safety and efficacy of an oral histone deacetylase inhibitor in systemic-onset juvenile idiopathic arthritis
- (2011) Jelena Vojinovic et al. ARTHRITIS AND RHEUMATISM
- Slowing Down Differentiation of Engrafted Human Myoblasts Into Immunodeficient Mice Correlates With Increased Proliferation and Migration
- (2011) Ingo Riederer et al. MOLECULAR THERAPY
- Stem Cell-Mediated Transfer of a Human Artificial Chromosome Ameliorates Muscular Dystrophy
- (2011) F. S. Tedesco et al. Science Translational Medicine
- Gentamicin-induced readthrough of stop codons in Duchenne muscular dystrophy
- (2010) Vinod Malik et al. ANNALS OF NEUROLOGY
- RNA Targeting Therapeutics: Molecular Mechanisms of Antisense Oligonucleotides as a Therapeutic Platform
- (2010) C. Frank Bennett et al. Annual Review of Pharmacology and Toxicology
- Identification and characterization of a non-satellite cell muscle resident progenitor during postnatal development
- (2010) Kathryn J. Mitchell et al. NATURE CELL BIOLOGY
- Dystrophin Immunity in Duchenne's Muscular Dystrophy
- (2010) Jerry R. Mendell et al. NEW ENGLAND JOURNAL OF MEDICINE
- Spliceosome Structure and Function
- (2010) C. L. Will et al. Cold Spring Harbor Perspectives in Biology
- Follow-up of three patients with a large in-frame deletion of exons 45–55 in the Duchenne muscular dystrophy (DMD) gene
- (2008) Akinori Nakamura et al. JOURNAL OF CLINICAL NEUROSCIENCE
Discover Peeref hubs
Discuss science. Find collaborators. Network.
Join a conversationAdd your recorded webinar
Do you already have a recorded webinar? Grow your audience and get more views by easily listing your recording on Peeref.
Upload Now