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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. 275 - 292
Exon-skipping therapy is an emerging approach that uses synthetic DNA-like molecules called antisense oligonucleotides (ASOs) to splice out frame-disrupting... 
mdx | mdx52 | Exon skipping | Duchenne muscular dystrophy (DMD) | Eteplirsen (Exondys 51) | Vivo-morpholinos (vPMOs) | Dystrophin | Multiexon skipping | Becker muscular dystrophy (BMD) | Phosphorodiamidate morpholino oligomer (PMO) | Index Medicus
Journal Article
Methods in Molecular Biology, ISSN 1064-3745, 2018, Volume 1828, pp. 365 - 379
Journal Article
Methods in Molecular Biology, ISSN 1064-3745, 2018, Volume 1828, pp. 79 - 90
Journal Article
Methods in Molecular Biology, ISSN 1064-3745, 2018, Volume 1828, pp. 497 - 502
Fibrodysplasia ossificans progressiva (FOP) is a rare autosomal-dominant disorder characterized by progressive heterotopic ossification. More than 95% of cases... 
Palovarotene | Bone morphogenetic protein (BMP) pathway | Musculoskeletal disorder | Exon skipping | Rapamycin | Retinoic acid receptor gamma (RARγ) selective agonist | ACVR1/ALK2 | Eteplirsen (Exondys 51) | Morpholino | Fibrodysplasia ossificans progressiva (FOP) | Index Medicus
Journal Article
Journal of neuromuscular diseases, ISSN 2214-3599, 2019, Volume 6, Issue 2, p. 213
Duchenne muscular dystrophy (DMD) patients experience skeletal muscle degeneration, including respiratory muscles. Respiratory decline in... 
Studies | Glucocorticoids | Duchenne's muscular dystrophy | Clinical trials | Degeneration | Dystrophy | Muscular dystrophy | Skeletal muscle
Journal Article
Journal of Neuromuscular Diseases, ISSN 2214-3599, 2018, Volume 5, Issue 1, pp. 47 - 58
Duchenne muscular dystrophy (DMD) is a rare, degenerative, X-linked genetic disease that results in progressive muscle loss and premature death, most commonly... 
Exondys 51 | vital capacity | dystrophin | maximal expiratory pressure | maximal inspiratory pressure | Cardiac muscle | Lung | Duchenne's muscular dystrophy | mRNA | Dystrophy | Mutation | Respiration | Dystrophin | Pressure | Heart diseases | Muscular dystrophy | Natural history
Journal Article
Methods in Molecular Biology, ISSN 1064-3745, 2018, Volume 1828, pp. 31 - 55
Exon skipping is a therapeutic approach that is feasible for various genetic diseases and has been studied and developed for over two decades. This approach... 
CRISPR Cas9/Cpf1 genome editing | Laminin-alpha 2 | Duchenne/Becker muscular dystrophy (DMD/BMD) | Dystrophic epidermolysis bullosa (DEB) | Myotonic dystrophy | Exon skipping | Usher syndrome | Eteplirsen (Exondys 51) | Fibrodysplasia ossificans progressiva (FOP) | Nusinersen (Spinraza) | Index Medicus
Journal Article
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