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The Application of Clinical Genetics, ISSN 1178-704X, 01/2019, Volume 12, pp. 113 - 130
Congenital muscular dystrophy (CMD) is a class of severe early-onset muscular dystrophies affecting skeletal/cardiac muscles as well as the central nervous... 
Pathology | Musculoskeletal system | Brain research | Congenital diseases | Scoliosis | MicroRNAs | Extracellular matrix | Nervous system | Mutation | Polyamines | Muscular dystrophy | phosphorodiamidate morpholino oligomer (PMO) | genome editing | LAMA2 | CRISPR/Cas9 | exon skipping | non-homologous end joining (NHEJ)
Journal Article
Methods in Molecular Biology, ISSN 1064-3745, 2018, Volume 1828, pp. 127 - 139
Exon skipping through the use of antisense oligonucleotides (AOs) is currently one of the most promising approaches for treating Duchenne muscular dystrophy... 
Clinical trial candidates | Immortalized DMD patient muscle cells | Dystrophin quantification | Duchenne/Becker muscular dystrophy (DMD/BMD) | Golodirsen | Eteplirsen | Human telomerase reverse transcriptase (hTERT) | Cyclin-dependent-kinase 4 (Cdk4) | NS-065/NCNP-01 | Primary muscle cells | Index Medicus
Journal Article
Proceedings of the National Academy of Sciences of the United States of America, ISSN 0027-8424, 04/2017, Volume 114, Issue 16, pp. 4213 - 4218
Duchenne muscular dystrophy (DMD) is a lethal genetic disorder caused by an absence of the dystrophin protein in bodywide muscles, including the heart.... 
Cardiac Purkinje fibers | Dystrophic dog model | Peptide-conjugated morpholinos | Duchenne muscular dystrophy | Exon skipping | MUSCLE PATHOLOGY | MULTIDISCIPLINARY SCIENCES | DMD GENE | CARDIOMYOPATHY | BODYWIDE | dystrophic dog model | peptide-conjugated morpholinos | MDX52 MICE | OLIGONUCLEOTIDES | RESCUE | exon skipping | LABORATORY-ANIMALS | cardiac Purkinje fibers | EXPRESSION | EXONS 45-55 | Genetic Therapy | Muscular Dystrophy, Animal - genetics | Exons | Male | Muscle, Skeletal - metabolism | Muscular Dystrophy, Animal - complications | Muscular Dystrophy, Duchenne - complications | Cardiomyopathies - etiology | Cardiomyopathies - therapy | Cell-Penetrating Peptides - pharmacology | Animals | Dogs | Morpholinos - pharmacology | Female | Muscular Dystrophy, Animal - therapy | Muscle, Skeletal - pathology | Muscular Dystrophy, Duchenne - genetics | Muscular Dystrophy, Duchenne - therapy | Dystrophin - metabolism | Disease Models, Animal | Heart | Physiological aspects | Health aspects | Conduction | Intravenous administration | Peptides | Toxicity | Cardiomyopathy | Effects | Muscular dystrophy | Fibers | Oligomers | Nerve conduction | Arginine | Duchenne's muscular dystrophy | Degeneration | Polymers | Heart diseases | Dystrophin | EKG | Cardiac muscle | Abnormalities | Muscles | Skeletal muscle | Purkinje fibers | Myocardium | Dystrophy | Index Medicus | Biological Sciences
Journal Article
Journal Article
Molecular Therapy, ISSN 1525-0016, 11/2017, Volume 25, Issue 11, pp. 2561 - 2572
Duchenne muscular dystrophy (DMD), the most common lethal genetic disorder, is caused by mutations in the ( ) gene. Exon skipping is a therapeutic approach... 
mdx52 mice | Becker muscular dystrophy | Exondys 51 | eteplirsen | exon skipping | Duchenne muscular dystrophy | clinical trial candidate screening | BMD | antisense morpholino | drisapersen | machine learning | hDMD/Dmd-null mice | MEDICINE, RESEARCH & EXPERIMENTAL | DIAGNOSIS | DESIGN | EFFICACY | DMD GENE | RESTORATION | PHASE-2 | OLIGONUCLEOTIDES | THERAPY | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | GENETICS & HEREDITY | MICE | ETEPLIRSEN | Exons | Humans | Oligonucleotides, Antisense - metabolism | Male | Muscle, Skeletal - metabolism | Reading Frames | Recovery of Function | RNA Splicing | Female | Muscular Dystrophy, Duchenne - therapy | Dystrophin - metabolism | Disease Models, Animal | Gene Expression | Morpholinos - genetics | Mice, Transgenic | Muscular Dystrophy, Duchenne - pathology | Morpholinos - metabolism | Animals | Oligonucleotides, Antisense - genetics | Dystrophin - genetics | Mice | Muscular Dystrophy, Duchenne - metabolism | Muscle, Skeletal - pathology | Muscular Dystrophy, Duchenne - genetics | Mutation | Genetic Therapy - methods | Performance evaluation | Medical research | Statistical analysis | Splicing | Antisense oligonucleotides | Muscular dystrophy | Proteins | Musculoskeletal system | Protein folding | Efficiency | Exon skipping | Duchenne's muscular dystrophy | Protein expression | Muscle function | Dystrophy | Dystrophin | Evacuations & rescues | Index Medicus | hDMD | Dmd-null mice | Original
Journal Article
Methods in Molecular Biology, ISSN 1064-3745, 2018, Volume 1828, pp. 3 - 30
Since its discovery in 1977, much has been known about RNA splicing and how it plays a central role in human development, function, and, notably, disease.... 
Cryptic splice correction | Exon skipping/inclusion | Exon inclusion | Golodirsen (SRP-4053) | Isoform switching | Antisense oligonucleotides | Antisense therapy | NS-065/NCNP-01 | Pre-mRNA splicing | Antisense history | Index Medicus
Journal Article
Journal of Personalized Medicine, ISSN 2075-4426, 12/2018, Volume 8, Issue 4, p. 38
Journal Article
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