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。Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMDmdx rat model
Audrey Bourdon et al.
GENE THERAPY (2022)
After Patient Death, FDA Places Hold on Pfizer Duchenne Muscular Dystrophy Gene Therapy Trial
Alex Philippidis
HUMAN GENE THERAPY (2022)
Control of backbone chemistry and chirality boost oligonucleotide splice switching activity
Pachamuthu Kandasamy et al.
NUCLEIC ACIDS RESEARCH (2022)
Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial
Laurent Servais et al.
NUCLEIC ACID THERAPEUTICS (2022)
Partial Restoration of Brain Dystrophin and Behavioral Deficits by Exon Skipping in the Muscular Dystrophy X-Linked (mdx) Mouse
Faouzi Zarrouki et al.
ANNALS OF NEUROLOGY (2022)
Pfizer Eyes Resuming Phase III Enrollment, Investigates Phase Ib Death Tied to Duchenne Muscular Dystrophy Candidate
Alex Philippidis
HUMAN GENE THERAPY (2022)
Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial
Craig M McDonald et al.
LANCET (2022)
Time to diagnosis of Duchenne muscular dystrophy remains unchanged: Findings from the Muscular Dystrophy Surveillance, Tracking, and Research Network, 2000-2015
Shiny Thomas et al.
MUSCLE & NERVE (2022)
Dilated cardiomyopathy as the initial presentation of Becker muscular dystrophy: a systematic review of published cases
Gaspar Del Rio-Pertuz et al.
ORPHANET JOURNAL OF RARE DISEASES (2022)
Histone deacetylase inhibitors improve antisense-mediated exon-skipping efficacy in mdx mice
Flavien Bizot et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2022)
Enhanced exon skipping and prolonged dystrophin restoration achieved by TfR1-targeted delivery of antisense oligonucleotide using FORCE conjugation in mdx mice
Cody A. Desjardins et al.
NUCLEIC ACIDS RESEARCH (2022)
Muscle regeneration affects Adeno Associated Virus 1 mediated transgene transcription
Amedee Mollard et al.
SCIENTIFIC REPORTS (2022)
Emerging therapies for Duchenne muscular dystrophy
Theodora Markati et al.
LANCET NEUROLOGY (2022)
Development of a novel, EEV-Conjugated PMO for Duchenne muscular dystrophy
N. Kreher et al.
NEUROMUSCULAR DISORDERS (2022)
Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMD
Britt Hanson et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2022)
Exon skipping induces uniform dystrophin rescue with dose-dependent restoration of serum miRNA biomarkers and muscle biophysical properties
Katarzyna Chwalenia et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2022)
Comparison of Long-term Ambulatory Function in Patients with Duchenne Muscular Dystrophy Treated with Eteplirsen and Matched Natural History Controls
Jerry R. Mendell et al.
JOURNAL OF NEUROMUSCULAR DISEASES (2021)
Dose-Escalation Study of Systemically Delivered rAAVrh74.MHCK7.micro-dystrophin in the mdx Mouse Model of Duchenne Muscular Dystrophy
Rachael A. Potter et al.
HUMAN GENE THERAPY (2021)
Molecular correction of Duchenne muscular dystrophy by splice modulation and gene editing
Britt Hanson et al.
RNA BIOLOGY (2021)
Assessment of rAAVrh.74.MHCK7.micro-dystrophin Gene Therapy Using Magnetic Resonance Imaging in Children With Duchenne Muscular Dystrophy
Rebecca J. Willcocks et al.
JAMA NETWORK OPEN (2021)
A Genotype-Phenotype Correlation Study of Exon Skip-Equivalent In-Frame Deletions and Exon Skip-Amenable Out-of-Frame Deletions across the DMD Gene to Simulate the Effects of Exon-Skipping Therapies: A Meta-Analysis
Saeed Anwar et al.
JOURNAL OF PERSONALIZED MEDICINE (2021)
Epigenetic Silencing of Recombinant Adeno-associated Virus Genomes by NP220 and the HUSH Complex
Anshuman Das et al.
JOURNAL OF VIROLOGY (2021)
Standard of care versus new-wave corticosteroids in the treatment of Duchenne muscular dystrophy: Can we do better?
Stephanie Kourakis et al.
ORPHANET JOURNAL OF RARE DISEASES (2021)
Improved transduction of canine X-linked muscular dystrophy with rAAV9-microdystrophin via multipotent MSC pretreatment
Hiromi Hayashita-Kinoh et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2021)
Lessons Learned from Discontinued Clinical Developments in Duchenne Muscular Dystrophy
Theodora Markati et al.
FRONTIERS IN PHARMACOLOGY (2021)
Casimersen: First Approval
Matt Shirley
DRUGS (2021)
Precise correction of Duchenne muscular dystrophy exon deletion mutations by base and prime editing
F. Chemello et al.
SCIENCE ADVANCES (2021)
The Effect of Immunomodulatory Treatments on Anti-Dystrophin Immune Response After AAV Gene Therapy in Dystrophin Deficient mdx Mice
Ning Li et al.
JOURNAL OF NEUROMUSCULAR DISEASES (2021)
Efficient precise in vivo base editing in adult dystrophic mice
Li Xu et al.
NATURE COMMUNICATIONS (2021)
Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping
Tabatha R. Simmons et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2021)
Renadirsen, a Novel 2′OMeRNA/ENA® Chimera Antisense Oligonucleotide, Induces Robust Exon 45 Skipping for Dystrophin In Vivo
Kentaro Ito et al.
CURRENT ISSUES IN MOLECULAR BIOLOGY (2021)
Chemical Proteomics and Phenotypic Profiling Identifies the Aryl Hydrocarbon Receptor as a Molecular Target of the Utrophin Modulator Ezutromid
Isabel V. L. Wilkinson et al.
ANGEWANDTE CHEMIE-INTERNATIONAL EDITION (2020)
Uniform sarcolemmal dystrophin expression is required to prevent extracellular microRNA release and improve dystrophic pathology
Tirsa L. E. van Westering et al.
JOURNAL OF CACHEXIA SARCOPENIA AND MUSCLE (2020)
Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping A Phase 2 Randomized Clinical Trial
Paula R. Clemens et al.
JAMA NEUROLOGY (2020)
Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy A Nonrandomized Controlled Trial
Jerry R. Mendell et al.
JAMA NEUROLOGY (2020)
Advances in oligonucleotide drug delivery
Thomas C. Roberts et al.
NATURE REVIEWS DRUG DISCOVERY (2020)
Mutation-independent Proteomic Signatures of Pathological Progression in Murine Models of Duchenne Muscular Dystrophy
Tirsa L. E. van Westering et al.
MOLECULAR & CELLULAR PROTEOMICS (2020)
Base editing: advances and therapeutic opportunities
Elizabeth M. Porto et al.
NATURE REVIEWS DRUG DISCOVERY (2020)
Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy
Craig Campbell et al.
JOURNAL OF COMPARATIVE EFFECTIVENESS RESEARCH (2020)
Genome Editing-Mediated Utrophin Upregulation in Duchenne Muscular Dystrophy Stem Cells
Kasturi Sengupta et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2020)
In Vivo Gene Editing of Muscle Stem Cells with Adeno-Associated Viral Vectors in a Mouse Model of Duchenne Muscular Dystrophy
Jennifer B. Kwon et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2020)
The potential of utrophin and dystrophin combination therapies for Duchenne muscular dystrophy
Simon Guiraud et al.
HUMAN MOLECULAR GENETICS (2019)
Identification of preexisting adaptive immunity to Cas9 proteins in humans
Carsten T. Charlesworth et al.
NATURE MEDICINE (2019)
Cardiac and skeletal muscle effects in the randomized HOPE-Duchenne trial
Michael Taylor et al.
NEUROLOGY (2019)
AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice
Michael E. Nance et al.
MOLECULAR THERAPY (2019)
Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function
Eric P. Hoffman et al.
NEUROLOGY (2019)
Potential Therapies Using Myogenic Stem Cells Combined with Bio-Engineering Approaches for Treatment of Muscular Dystrophies
Norio Motohashi et al.
CELLS (2019)
Search-and-replace genome editing without double-strand breaks or donor DNA
Andrew V. Anzalone et al.
NATURE (2019)
Non-immunogenic utrophin gene therapy for the treatment of muscular dystrophy animal models
Yafeng Song et al.
NATURE MEDICINE (2019)
Clinical Evidence Supporting Early Treatment Of Patients With Spinal Muscular Atrophy: Current Perspectives
Tamara Dangouloff et al.
THERAPEUTICS AND CLINICAL RISK MANAGEMENT (2019)
High prevalence of Streptococcus pyogenes Cas9-reactive T cells within the adult human population
Dimitrios L. Wagner et al.
NATURE MEDICINE (2019)
A Sequel to the Eteplirsen Saga: Eteplirsen Is Approved in the United States but Was Not Approved in Europe
Annemieke Aartsma-Rus et al.
NUCLEIC ACID THERAPEUTICS (2019)
A Phase 1b Trial to Assess the Pharmacokinetics of Ezutromid in Pediatric Duchenne Muscular Dystrophy Patients on a Balanced Diet
Francesco Muntoni et al.
CLINICAL PHARMACOLOGY IN DRUG DEVELOPMENT (2019)
Utrophin up-regulation by artificial transcription factors induces muscle rescue and impacts the neuromuscular junction in mdx mice
Cinzia Pisani et al.
BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE (2018)
Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN
Christian Hinderer et al.
HUMAN GENE THERAPY (2018)
Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management
David J. Birnkrant et al.
LANCET NEUROLOGY (2018)
Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy
Dongsheng Duan
MOLECULAR THERAPY (2018)
Adenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophy
Seuk-Min Ryu et al.
NATURE BIOTECHNOLOGY (2018)
Eteplirsen treatment for Duchenne muscular dystrophy Exon skipping and dystrophin production
Jay S. Charleston et al.
NEUROLOGY (2018)
Systemic administration of the antisense oligonucleotide NS-065/NCNP-01 for skipping of exon 53 in patients with Duchenne muscular dystrophy
Hirofumi Komaki et al.
SCIENCE TRANSLATIONAL MEDICINE (2018)
High-Yield Purification, Preservation, and Serial Transplantation of Human Satellite Cells
Steven M. Garcia et al.
STEM CELL REPORTS (2018)
Combined Therapies for Duchenne Muscular Dystrophy to Optimize Treatment Efficacy
Gonzalo Cordova et al.
FRONTIERS IN GENETICS (2018)
Prevalence of Pre-existing Antibodies to CRISPR-Associated Nuclease Cas9 in the USA Population
Vijaya L. Simhadri et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2018)
Genomic analysis identifies frequent deletions of Dystrophin in olfactory neuroblastoma
Gary L. Gallia et al.
NATURE COMMUNICATIONS (2018)
Adverse effects of a single dose of gentamicin in adults: a systematic review
Rachel S. Hayward et al.
BRITISH JOURNAL OF CLINICAL PHARMACOLOGY (2018)
Comparison of the phenotypes of patients harboring in-frame deletions starting at exon 45 in the Duchenne muscular dystrophy gene indicates potential for the development of exon skipping therapy
Akinori Nakamura et al.
JOURNAL OF HUMAN GENETICS (2017)
Comparison of Serum rAAV Serotype-Specific Antibodies in Patients with Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Inclusion Body Myositis, or GNE Myopathy
Deborah A. Zygmunt et al.
HUMAN GENE THERAPY (2017)
Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
Craig M. McDonald et al.
LANCET (2017)
Duchenne drug clings on for FDA nod
[Anonymous]
NATURE BIOTECHNOLOGY (2017)
Response to Railroading at the FDA
Francesco Muntoni et al.
NATURE BIOTECHNOLOGY (2017)
Control of phosphorothioate stereochemistry substantially increases the efficacy of antisense oligonucleotides
Naoki Iwamoto et al.
NATURE BIOTECHNOLOGY (2017)
Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy
Leonela Amoasii et al.
SCIENCE TRANSLATIONAL MEDICINE (2017)
Downregulation of myostatin pathway in neuromuscular diseases may explain challenges of anti-myostatin therapeutic approaches
Virginie Mariot et al.
NATURE COMMUNICATIONS (2017)
Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy
Caroline Le Guiner et al.
NATURE COMMUNICATIONS (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)
FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga
Annemieke Aartsma-Rus et al.
NUCLEIC ACID THERAPEUTICS (2017)
CRISPR-Cpf1 correction of muscular dystrophy mutations in human cardiomyocytes and mice
Yu Zhang et al.
SCIENCE ADVANCES (2017)
Functional improvement of dystrophic muscle by repression of utrophin: let-7c interaction
Manoj K. Mishra et al.
PLOS ONE (2017)
In Vivo Target Gene Activation via CRISPR/Cas9-Mediated Trans-epigenetic Modulation
Hsin-Kai Liao et al.
CELL (2017)
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)
Relation of Cardiac Dysfunction to Rhythm Abnormalities in Patients With Duchenne or Becker Muscular Dystrophies
David Y. Chiang et al.
AMERICAN JOURNAL OF CARDIOLOGY (2016)
Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy
Jerry R. Mendell et al.
ANNALS OF NEUROLOGY (2016)
A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells
Courtney S. Young et al.
CELL STEM CELL (2016)
Antisense pre-treatment increases gene therapy efficacy in dystrophic muscles
Cecile Peccate et al.
HUMAN MOLECULAR GENETICS (2016)
Deletion of exons 3-9 encompassing a mutational hot spot in the DMD gene presents an asymptomatic phenotype, indicating a target region for multiexon skipping therapy
Akinori Nakamura et al.
JOURNAL OF HUMAN GENETICS (2016)
Railroading at the FDA
[Anonymous]
NATURE BIOTECHNOLOGY (2016)
Eteplirsen therapy for Duchenne muscular dystrophy: skipping to the front of the line
James J. Dowling
NATURE REVIEWS NEUROLOGY (2016)
Histological effects of givinostat in boys with Duchenne muscular dystrophy
Paolo Bettica et al.
NEUROMUSCULAR DISORDERS (2016)
Intra-arterial transplantation of HLA-matched donor mesoangioblasts in Duchenne muscular dystrophy
Giulio Cossu et al.
EMBO MOLECULAR MEDICINE (2015)
Multi-level omics analysis in a murine model of dystrophin loss and therapeutic restoration
Thomas C. Roberts et al.
HUMAN MOLECULAR GENETICS (2015)
How much dystrophin is enough: the physiological consequences of different levels of dystrophin in the mdx mouse
Caroline Godfrey et al.
HUMAN MOLECULAR GENETICS (2015)
Safe and bodywide muscle transduction in young adult Duchenne muscular dystrophy dogs with adeno-associated virus
Yongping Yue et al.
HUMAN MOLECULAR GENETICS (2015)
Second-generation compound for the modulation of utrophin in the therapy of DMD
Simon Guiraud et al.
HUMAN MOLECULAR GENETICS (2015)
The TREAT-NMD DMD Global Database: Analysis of More than 7,000 Duchenne Muscular Dystrophy Mutations
Catherine L. Bladen et al.
HUMAN MUTATION (2015)
Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers
Aurelie Goyenvalle et al.
NATURE MEDICINE (2015)
Dystrophin expression in muscle stem cells regulates their polarity and asymmetric division
Nicolas A. Dumont et al.
NATURE MEDICINE (2015)
Satellite cells from dystrophic muscle retain regenerative capacity
Luisa Boldrin et al.
STEM CELL RESEARCH (2015)
Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy
David G. Ousterout et al.
NATURE COMMUNICATIONS (2015)
Prevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice
Corinne A. Betts et al.
SCIENTIFIC REPORTS (2015)
TNF-α-Induced microRNAs Control Dystrophin Expression in Becker Muscular Dystrophy
Alyson A. Fiorillo et al.
CELL REPORTS (2015)
The effect of the muscle environment on the regenerative capacity of human skeletal muscle stem cells
Jinhong Meng et al.
SKELETAL MUSCLE (2015)
Dystrophin levels and clinical severity in Becker muscular dystrophy patients
J. C. van den Bergen et al.
JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY (2014)
Vascular Delivery of rAAVrh74.MCK.GALGT2 to the Gastrocnemius Muscle of the Rhesus Macaque Stimulates the Expression of Dystrophin and Laminin α2 Surrogates
Louis G. Chicoine et al.
MOLECULAR THERAPY (2014)
ATALUREN TREATMENT OF PATIENTS WITH NONSENSE MUTATION DYSTROPHINOPATHY
Katharine Bushby et al.
MUSCLE & NERVE (2014)
Dystrophin is a tumor suppressor in human cancers with myogenic programs
Yuexiang Wang et al.
NATURE GENETICS (2014)
Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice
Nicolas Wein et al.
NATURE MEDICINE (2014)
Synthesis, biophysical properties and biological activity of second generation antisense oligonucleotides containing chiral phosphorothioate linkages
W. Brad Wan et al.
NUCLEIC ACIDS RESEARCH (2014)
Microtubule binding distinguishes dystrophin from utrophin
Joseph J. Belanto et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2014)
Biochemical Characterization of Patients With In-Frame or Out-of-Frame DMD Deletions Pertinent to Exon 44 or 45 Skipping
Karen Anthony et al.
JAMA NEUROLOGY (2014)
Adeno-associated viral vectors do not efficiently target muscle satellite cells
Andrea L. H. Arnett et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2014)
Eteplirsen for the Treatment of Duchenne Muscular Dystrophy
Jerry R. Mendell et al.
ANNALS OF NEUROLOGY (2013)
CRISPR-Mediated Modular RNA-Guided Regulation of Transcription in Eukaryotes
Luke A. Gilbert et al.
CELL (2013)
Micro-dystrophin and follistatin co-delivery restores muscle function in aged DMD model
Louise R. Rodino-Klapac et al.
HUMAN MOLECULAR GENETICS (2013)
Long-term benefits and adverse effects of intermittent versus daily glucocorticoids in boys with Duchenne muscular dystrophy
Valeria Ricotti et al.
JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY (2013)
AAV Genome Loss From Dystrophic Mouse Muscles During AAV-U7 snRNA-mediated Exon-skipping Therapy
Maeva Le Hir et al.
MOLECULAR THERAPY (2013)
Mammalian Base Excision Repair: the Forgotten Archangel
Grigory L. Dianov et al.
NUCLEIC ACIDS RESEARCH (2013)
Multiplex Genome Engineering Using CRISPR/Cas Systems
Le Cong et al.
SCIENCE (2013)
Pre-existing Anti-Adeno-Associated Virus Antibodies as a Challenge in AAV Gene Therapy
Vedell Louis Jeune et al.
HUMAN GENE THERAPY METHODS (2013)
A Lack of Premature Termination Codon Read-Through Efficacy of PTC124 (Ataluren) in a Diverse Array of Reporter Assays
Stuart P. McElroy et al.
PLOS BIOLOGY (2013)
Prednisolone Treatment Does Not Interfere with 2′-O-Methyl Phosphorothioate Antisense-Mediated Exon Skipping in Duchenne Muscular Dystrophy
Ingrid E. C. Verhaart et al.
HUMAN GENE THERAPY (2012)
Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery
Yoshitsugu Aoki et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2012)
Dantrolene Enhances Antisense-Mediated Exon Skipping in Human and Mouse Models of Duchenne Muscular Dystrophy
Genevieve C. Kendall et al.
SCIENCE TRANSLATIONAL MEDICINE (2012)
Dual Myostatin and Dystrophin Exon Skipping by Morpholino Nucleic Acid Oligomers Conjugated to a Cell-penetrating Peptide Is a Promising Therapeutic Strategy for the Treatment of Duchenne Muscular Dystrophy
Alberto Malerba et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2012)
Expression Analysis in Multiple Muscle Groups and Serum Reveals Complexity in the MicroRNA Transcriptome of the mdx Mouse with Implications for Therapy
Thomas C. Roberts et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2012)
Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment
Corinne Betts et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2012)
miR-31 modulates dystrophin expression: new implications for Duchenne muscular dystrophy therapy
Davide Cacchiarelli et al.
EMBO REPORTS (2011)
Discovery of 2-Arylbenzoxazoles as Upregulators of Utrophin Production for the Treatment of Duchenne Muscular Dystrophy
Daniel R. Chancellor et al.
JOURNAL OF MEDICINAL CHEMISTRY (2011)
Histone Deacetylase Inhibitors in the Treatment of Muscular Dystrophies: Epigenetic Drugs for Genetic Diseases
Silvia Consalvi et al.
MOLECULAR MEDICINE (2011)
Duchenne muscular dystrophy: Survival by cardio-respiratory interventions
Yuka Ishikawa et al.
NEUROMUSCULAR DISORDERS (2011)
Daily Treatment with SMTC1100, a Novel Small Molecule Utrophin Upregulator, Dramatically Reduces the Dystrophic Symptoms in the mdx Mouse
Jonathon M. Tinsley et al.
PLOS ONE (2011)
Translational Regulation of Utrophin by miRNAs
Utpal Basu et al.
PLOS ONE (2011)
Morpholinos and their peptide conjugates: Therapeutic promise and challenge for Duchenne muscular dystrophy
Hong M. Moulton et al.
BIOCHIMICA ET BIOPHYSICA ACTA-BIOMEMBRANES (2010)
Dose-dependent restoration of dystrophin expression in cardiac muscle of dystrophic mice by systemically delivered morpholino
B. Wu et al.
GENE THERAPY (2010)
Sarcolemmal nNOS anchoring reveals a qualitative difference between dystrophin and utrophin
Dejia Li et al.
JOURNAL OF CELL SCIENCE (2010)
Combination of Myostatin Pathway Interference and Dystrophin Rescue Enhances Tetanic and Specific Force in Dystrophic mdx Mice
Julie Dumonceaux et al.
MOLECULAR THERAPY (2010)
Brief Report: Dystrophin Immunity in Duchenne's Muscular Dystrophy.
Jerry R. Mendell et al.
NEW ENGLAND JOURNAL OF MEDICINE (2010)
Molecular basis for the high-affinity binding and stabilization of firefly luciferase by PTC124
Douglas S. Auld et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2010)
Sarcoglycanopathies: molecular pathogenesis and therapeutic prospects
Dorianna Sandona et al.
EXPERT REVIEWS IN MOLECULAR MEDICINE (2009)
Common micro-RNA signature in skeletal muscle damage and regeneration induced by Duchenne muscular dystrophy and acute ischemia
Simona Greco et al.
FASEB JOURNAL (2009)
Theoretic Applicability of Antisense-Mediated Exon Skipping for Duchenne Muscular Dystrophy Mutations
Annemieke Aartsma-Rus et al.
HUMAN MUTATION (2009)
Adeno-associated virus vector Genomes persist as episomal chromatin in primate muscle
Magalie Penaud-Budloo et al.
JOURNAL OF VIROLOGY (2008)
Microutrophin delivery through rAAV6 increases lifespan and improves muscle function in dystrophic dystrophin/utrophin-deficient mice
Guy L. Odom et al.
MOLECULAR THERAPY (2008)
HDAC2 blockade by nitric oxide and histone deacetylase inhibitors reveals a common target in Duchenne muscular dystrophy treatment
Claudia Colussi et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2008)
Pharmacokinetics, biodistribution, stability and toxicity of a cell-penetrating peptide-morpholino oligomer conjugate
Adams Amantana et al.
BIOCONJUGATE CHEMISTRY (2007)
PTC124 targets genetic disorders caused by nonsense mutations
Ellen M. Welch et al.
NATURE (2007)
Hereditary myosin myopathies
Anders Oldfors
NEUROMUSCULAR DISORDERS (2007)
Pericytes of human skeletal muscle are myogenic precursors distinct from satellite cells
Arianna Dellavalle et al.
NATURE CELL BIOLOGY (2007)
Multiexon skipping leading to an artificial DMD protein lacking amino acids from exons 45 through 55 could rescue up to 63 % of patients with Duchenne muscular dystrophy
Christophe Beroud et al.
HUMAN MUTATION (2007)
Design of tissue-specific regulatory cassettes for high-level rAAV-mediated expression in skeletal and cardiac muscle
Maja Z. Salva et al.
MOLECULAR THERAPY (2007)
Autologous transplantation of muscle-derived CD133(+) stem cells in Duchenne muscle patients
Y. Torrente et al.
CELL TRANSPLANTATION (2007)
Dystrophin expression in muscles of Duchenne muscular dystrophy patients after high-density injections of normal myogenic cells
Daniel Skuk et al.
JOURNAL OF NEUROPATHOLOGY AND EXPERIMENTAL NEUROLOGY (2006)
Phenotypic improvement of dystrophic muscles by rAAV/microdystrophin vectors is augmented by Igf1 codelivery
S Abmayr et al.
MOLECULAR THERAPY (2005)
Long-term pharmacologically regulated expression of erythropoietin in,primates following AAV-mediated gene transfer
VM Rivera et al.
BLOOD (2005)
Systemic delivery of genes to striated muscles using adeno-associated viral vectors
P Gregorevic et al.
NATURE MEDICINE (2004)
Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping
A Goyenvalle et al.
SCIENCE (2004)
Dystroglycan, a scaffold for the ERK-MAP kinase cascade
HJ Spence et al.
EMBO REPORTS (2004)
Dystrophin and mutations: one gene, several proteins, multiple phenotypes
F Muntoni et al.
LANCET NEUROLOGY (2003)
Prevention of pathology in mdx mice by expression of utrophin:: analysis using an inducible transgenic expression system
S Squire et al.
HUMAN MOLECULAR GENETICS (2002)
Chimeric snRNA molecules carrying antisense sequences against the splice junctions of exon 51 of the dystrophin pre-mRNA induce exon skipping and restoration of a dystrophin synthesis in Δ48-50 DMD cells
FG De Angelis et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2002)
Non-toxic ubiquitous over-expression of utrophin in the mdx mouse
R Fisher et al.
NEUROMUSCULAR DISORDERS (2001)
Fracture risk in patients with muscular dystrophy and spinal muscular atrophy
P Vestergaard et al.
JOURNAL OF REHABILITATION MEDICINE (2001)
The dystrophin complex forms a mechanically strong link between the sarcolemma and costameric actin
IN Rybakova et al.
JOURNAL OF CELL BIOLOGY (2000)
Repeat transduction in the mouse lung by using adeno-associated virus vectors with different serotypes
CL Halbert et al.
JOURNAL OF VIROLOGY (2000)