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。Developments in reading frame restoring therapy approaches for Duchenne muscular dystrophy
Anne-Fleur E. Schneider et al.
EXPERT OPINION ON BIOLOGICAL THERAPY (2021)
Stem cell-based therapies for Duchenne muscular dystrophy
Congshan Sun et al.
EXPERIMENTAL NEUROLOGY (2020)
Dystrophin Dp71 and the Neuropathophysiology of Duchenne Muscular Dystrophy
Michael Naidoo et al.
MOLECULAR NEUROBIOLOGY (2020)
Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy
Diane E. Frank et al.
NEUROLOGY (2020)
Effect of Ataluren on dystrophin mutations
Joachim Berger et al.
JOURNAL OF CELLULAR AND MOLECULAR MEDICINE (2020)
Correction of muscular dystrophies by CRISPR gene editing
Francesco Chemello et al.
JOURNAL OF CLINICAL INVESTIGATION (2020)
The Genetic Landscape of Dystrophin Mutations in Italy: A Nationwide Study
Marcella Neri et al.
FRONTIERS IN GENETICS (2020)
Therapeutic Strategies for Duchenne Muscular Dystrophy: An Update
Chengmei Sun et al.
GENES (2020)
Muscle and cardiac therapeutic strategies for Duchenne muscular dystrophy: past, present, and future
Agnieszka Loboda et al.
PHARMACOLOGICAL REPORTS (2020)
Casimersen treatment in eligible patients with Duchenne muscular dystrophy: Safety, tolerability, and pharmacokinetics over 144 weeks of treatment
N. Kuntz et al.
NEUROMUSCULAR DISORDERS (2020)
Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy
Craig Campbell et al.
JOURNAL OF COMPARATIVE EFFECTIVENESS RESEARCH (2020)
Current Genetic Survey and Potential Gene-Targeting Therapeutics for Neuromuscular Diseases
Wei Chiu et al.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2020)
Viltolarsen in Japanese Duchenne muscular dystrophy patients: A phase 1/2 study
Hirofumi Komaki et al.
ANNALS OF CLINICAL AND TRANSLATIONAL NEUROLOGY (2020)
Novel genetic therapeutic approaches for modulating the severity of β-thalassemia (Review)
Fareeha Amjad et al.
BIOMEDICAL REPORTS (2020)
Stem cells, blood vessels, and angiogenesis as major determinants for musculoskeletal tissue repair
Johnny Huard
JOURNAL OF ORTHOPAEDIC RESEARCH (2019)
Non-invasive Prenatal Testing Using Fetal DNA
Giulia Breveglieri et al.
MOLECULAR DIAGNOSIS & THERAPY (2019)
Development of Novel Micro-dystrophins with Enhanced Functionality
Julian N. Ramos et al.
MOLECULAR THERAPY (2019)
Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy
Christopher E. Nelson et al.
NATURE MEDICINE (2019)
Cardiac and skeletal muscle effects in the randomized HOPE-Duchenne trial
Michael Taylor et al.
NEUROLOGY (2019)
CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells
Yi-Li Min et al.
SCIENCE ADVANCES (2019)
Evaluating the Impact of Variable Phosphorothioate Content in Tricyclo-DNA Antisense Oligonucleotides in a Duchenne Muscular Dystrophy Mouse Model
Lucia Echevarria et al.
NUCLEIC ACID THERAPEUTICS (2019)
AAV-based gene therapies for the muscular dystrophies
Julie M. Crudele et al.
HUMAN MOLECULAR GENETICS (2019)
Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseases
Maria K. Tsoumpra et al.
EBIOMEDICINE (2019)
CRISPR/Cas9-Based Dystrophin Restoration Reveals a Novel Role for Dystrophin in Bioenergetics and Stress Resistance of Muscle Progenitors
Polina R. Matre et al.
STEM CELLS (2019)
Dp71 Expression in Human Glioblastoma
Simona Ruggieri et al.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2019)
Non-immunogenic utrophin gene therapy for the treatment of muscular dystrophy animal models
Yafeng Song et al.
NATURE MEDICINE (2019)
rAAVrh74.MCK.GALGT2 Protects against Loss of Hemodynamic Function in the Aging mdx Mouse Heart
Rui Xu et al.
MOLECULAR THERAPY (2019)
The Dystrophin Glycoprotein Complex Regulates the Epigenetic Activation of Muscle Stem Cell Commitment
Natasha C. Chang et al.
CELL STEM CELL (2018)
Recapitulating muscle disease phenotypes with myotonic dystrophy 1 induced pluripotent stem cells: a tool for disease modeling and drug discovery
Ricardo Mondragon-Gonzalez et al.
DISEASE MODELS & MECHANISMS (2018)
The roles of muscle stem cells in muscle injury, atrophy and hypertrophy
So-ichiro Fukada
JOURNAL OF BIOCHEMISTRY (2018)
Descriptive Phenotype of Obsessive Compulsive Symptoms in Males With Duchenne Muscular Dystrophy
Angela J. Lee et al.
JOURNAL OF CHILD NEUROLOGY (2018)
Experiences of Women Who Have Had Carrier Testing for Duchenne Muscular Dystrophy and Becker Muscular Dystrophy During Adolescence
Harry G. Fraser et al.
JOURNAL OF GENETIC COUNSELING (2018)
Altered membrane integrity in the progression of muscle diseases
Kevin E. McElhanon et al.
LIFE SCIENCES (2018)
A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy
Nathalie Goemans et al.
NEUROMUSCULAR DISORDERS (2018)
Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle (vol 8, 2017)
James S. Novak et al.
NATURE COMMUNICATIONS (2018)
Three-Dimensional Human iPSC-Derived Artificial Skeletal Muscles Model Muscular Dystrophies and Enable Multilineage Tissue Engineering
Sara Martina Maffioletti et al.
CELL REPORTS (2018)
Saponins as Natural Adjuvant for Antisense Morpholino Oligonucleotides Delivery In Vitro and in mdx Mice
Mingxing Wang et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2018)
Placebo-controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy
Craig M. McDonald et al.
ANNALS OF CLINICAL AND TRANSLATIONAL NEUROLOGY (2018)
Combined Therapies for Duchenne Muscular Dystrophy to Optimize Treatment Efficacy
Gonzalo Cordova et al.
FRONTIERS IN GENETICS (2018)
Personalized gene and cell therapy for Duchenne Muscular Dystrophy
Florian Barthelemy et al.
NEUROMUSCULAR DISORDERS (2018)
Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy
Leonela Amoasii et al.
SCIENCE (2018)
Emerging Strategies in the Treatment of Duchenne Muscular Dystrophy
Perry B. Shieh
NEUROTHERAPEUTICS (2018)
AAV CRISPR editing rescues cardiac and muscle function for 18 months in dystrophic mice
Chady H. Hakim et al.
JCI INSIGHT (2018)
Antisense Oligonucleotide-Based Therapy for Neuromuscular Disease
Valentina Sardone et al.
MOLECULES (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)
A human iPS cell myogenic differentiation system permitting high-throughput drug screening
Tomoya Uchimura et al.
STEM CELL RESEARCH (2017)
Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy
Caroline Le Guiner et al.
NATURE COMMUNICATIONS (2017)
Next-generation mapping: a novel approach for detection of pathogenic structural variants with a potential utility in clinical diagnosis
Hayk Barseghyan et al.
GENOME MEDICINE (2017)
Timing and localization of human dystrophin isoform expression provide insights into the cognitive phenotype of Duchenne muscular dystrophy
Nathalie Doorenweerd et al.
SCIENTIFIC REPORTS (2017)
In Vivo Genome Editing Restores Dystrophin Expression and Cardiac Function in Dystrophic Mice
Mona El Refaey et al.
CIRCULATION RESEARCH (2017)
Pharmacological advances for treatment in Duchenne muscular dystrophy
Simon Guiraud et al.
CURRENT OPINION IN PHARMACOLOGY (2017)
Neurodevelopmental, emotional, and behavioural problems in Duchenne muscular dystrophy in relation to underlying dystrophin gene mutations
Valeria Ricotti et al.
DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY (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)
B4GALNT2 (GALGT2) Gene Therapy Reduces Skeletal Muscle Pathology in the FKRP P448L Mouse Model of Limb Girdle Muscular Dystrophy 21
Paul J. Thomas et al.
AMERICAN JOURNAL OF PATHOLOGY (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)
A multifunctional AAV-CRISPR-Cas9 and its host response
Wei Leong Chew et al.
NATURE METHODS (2016)
Concordant but Varied Phenotypes among Duchenne Muscular Dystrophy Patient-Specific Myoblasts Derived using a Human iPSC-Based Model
In Young Choi et al.
CELL REPORTS (2016)
Pharmacological therapeutics targeting the secondary defects and downstream pathology of Duchenne muscular dystrophy
Janelle M. Spinazzola et al.
EXPERT OPINION ON ORPHAN DRUGS (2016)
Intra-arterial transplantation of HLA-matched donor mesoangioblasts in Duchenne muscular dystrophy
Giulio Cossu et al.
EMBO MOLECULAR MEDICINE (2015)
Second-generation compound for the modulation of utrophin in the therapy of DMD
Simon Guiraud et al.
HUMAN MOLECULAR GENETICS (2015)
Duchenne Muscular Dystrophy: From Diagnosis to Therapy
Maria Sofia Falzarano et al.
MOLECULES (2015)
Therapeutic genome editing: prospects and challenges
David Benjamin Turitz Cox 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)
Dystrophin-glycoprotein complex regulates muscle nitric oxide production through mechanoregulation of AMPK signaling
Joanne F. Garbincius et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2015)
Satellite cells from dystrophic muscle retain regenerative capacity
Luisa Boldrin et al.
STEM CELL RESEARCH (2015)
Cancer Stem Cell Division: When the Rules of Asymmetry Are Broken
Subhas Mukherjee et al.
STEM CELLS AND DEVELOPMENT (2015)
Early pathogenesis of Duchenne muscular dystrophy modelled in patient-derived human induced pluripotent stem cells
Emi Shoji et al.
SCIENTIFIC REPORTS (2015)
The Dystrophin Complex: Structure, Function, and Implications for Therapy
Quan Q. Gao et al.
COMPREHENSIVE PHYSIOLOGY (2015)
Analysis of off-target effects of CRISPR/Cas-derived RNA-guided endonucleases and nickases
Seung Woo Cho et al.
GENOME RESEARCH (2014)
Nitric oxide synthase deficiency and the pathophysiology of muscular dystrophy
James G. Tidball et al.
JOURNAL OF PHYSIOLOGY-LONDON (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)
Improving CRISPR-Cas nuclease specificity using truncated guide RNAs
Yanfang Fu et al.
NATURE BIOTECHNOLOGY (2014)
CRISPR-Cas systems for editing, regulating and targeting genomes
Jeffry D. Sander et al.
NATURE BIOTECHNOLOGY (2014)
Dystrophin is a tumor suppressor in human cancers with myogenic programs
Yuexiang Wang et al.
NATURE GENETICS (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)
Seamless modification of wild-type induced pluripotent stem cells to the natural CCR5Δ32 mutation confers resistance to HIV infection
Lin Ye et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2014)
Myogenic Differentiation of Muscular Dystrophy-Specific Induced Pluripotent Stem Cells for Use in Drug Discovery
Ramzey Abujarour et al.
STEM CELLS TRANSLATIONAL MEDICINE (2014)
ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering
Thomas Gaj et al.
TRENDS IN BIOTECHNOLOGY (2013)
Molecular and functional heterogeneity of GABAergic synapses
Jean-Marc Fritschy et al.
CELLULAR AND MOLECULAR LIFE SCIENCES (2012)
A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity
Martin Jinek et al.
SCIENCE (2012)
Restoring Dystrophin Expression in Duchenne Muscular Dystrophy Muscle Progress in Exon Skipping and Stop Codon Read Through
Eric P. Hoffman et al.
AMERICAN JOURNAL OF PATHOLOGY (2011)
The genome of self-complementary adeno-associated viral vectors increases Toll-like receptor 9-dependent innate immune responses in the liver
Ashley T. Martino et al.
BLOOD (2011)
Latest developments in the large-scale production of adeno-associated virus vectors in insect cells toward the treatment of neuromuscular diseases
Lionel Galibert et al.
JOURNAL OF INVERTEBRATE PATHOLOGY (2011)
A TALE nuclease architecture for efficient genome editing
Jeffrey C. Miller et al.
NATURE BIOTECHNOLOGY (2011)
Voltage-Gated Ion Channel Dysfunction Precedes Cardiomyopathy Development in the Dystrophic Heart
Xaver Koenig et al.
PLOS ONE (2011)
Drug Discovery for Duchenne Muscular Dystrophy via Utrophin Promoter Activation Screening
Catherine Moorwood et al.
PLOS ONE (2011)
Neurocognitive Profiles in Duchenne Muscular Dystrophy and Gene Mutation Site
Maria Grazia D'Angelo et al.
PEDIATRIC NEUROLOGY (2011)
Pericytes resident in postnatal skeletal muscle differentiate into muscle fibres and generate satellite cells
A. Dellavalle et al.
NATURE COMMUNICATIONS (2011)
Widespread Muscle Expression of an AAV9 Human Mini-dystrophin Vector After Intravenous Injection in Neonatal Dystrophin-deficient Dogs
Joe N. Kornegay et al.
MOLECULAR THERAPY (2010)
Genome editing with engineered zinc finger nucleases
Fyodor D. Urnov et al.
NATURE REVIEWS GENETICS (2010)
Revertant fibres and dystrophin traces in Duchenne muscular dystrophy: Implication for clinical trials
Virginia Arechavala-Gomeza et al.
NEUROMUSCULAR DISORDERS (2010)
Brief Report: Dystrophin Immunity in Duchenne's Muscular Dystrophy.
Jerry R. Mendell et al.
NEW ENGLAND JOURNAL OF MEDICINE (2010)
Dystrophin Gene Mutation Location and the Risk of Cognitive Impairment in Duchenne Muscular Dystrophy
Peter J. Taylor et al.
PLOS ONE (2010)
Overexpression of Galgt2 in skeletal muscle prevents injury resulting from eccentric contractions in both mdx and wild-type mice
Paul T. Martin et al.
AMERICAN JOURNAL OF PHYSIOLOGY-CELL PHYSIOLOGY (2009)
Theoretic Applicability of Antisense-Mediated Exon Skipping for Duchenne Muscular Dystrophy Mutations
Annemieke Aartsma-Rus et al.
HUMAN MUTATION (2009)
Role of Mental Retardation-Associated Dystrophin-Gene Product Dp71 in Excitatory Synapse Organization, Synaptic Plasticity and Behavioral Functions
Fatma Daoud et al.
PLOS ONE (2009)
Pericytes of human skeletal muscle are myogenic precursors distinct from satellite cells
Arianna Dellavalle et al.
NATURE CELL BIOLOGY (2007)
Mesoangioblast stem cells ameliorate muscle function in dystrophic dogs
Maurilio Sampaolesi et al.
NATURE (2006)
Cardiac sodium channel Nav1.5 is regulated by a multiprotein complex composed of syntrophins and dystrophin
Bruno Gavillet et al.
CIRCULATION RESEARCH (2006)
Entries in the Leiden Duchenne muscular dystrophy mutation database: An overview of mutation types and paradoxical cases that confirm the reading-frame rule
Annemieke Aartsma-Rus et al.
MUSCLE & NERVE (2006)
Sparks, signals and shock absorbers: how dystrophin loss causes muscular dystrophy
CL Batchelor et al.
TRENDS IN CELL BIOLOGY (2006)
Copy number variation in the genome; the human DMD gene as an example
S. J. White et al.
CYTOGENETIC AND GENOME RESEARCH (2006)
Cell therapy of α-sarcoglycan null dystrophic mice through intra-arterial delivery of mesoangioblasts
M Sampaolesi et al.
SCIENCE (2003)
A novel engineered meganuclease induces homologous recombination in yeast and mammalian cells
JC Epinat et al.
NUCLEIC ACIDS RESEARCH (2003)
Dystrophin and mutations: one gene, several proteins, multiple phenotypes
F Muntoni et al.
LANCET NEUROLOGY (2003)
Adeno-associated virus vector-mediated minidystrophin gene therapy improves dystrophic muscle contractile function in mdx mice
J Watchko et al.
HUMAN GENE THERAPY (2002)
Regulation of the cardiac L-type Ca2+ channel by the actin-binding proteins α-actinin and dystrophin
A Sadeghi et al.
AMERICAN JOURNAL OF PHYSIOLOGY-CELL PHYSIOLOGY (2002)
分享论文
