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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. v - vi
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 Human Genetics, ISSN 1434-5161, 03/2018, Volume 63, Issue 3, pp. 365 - 375
Oligonucleotide-mediated splicing modulation is a promising therapeutic approach for Duchenne muscular dystrophy (DMD). Recently, eteplirsen, a... 
DESIGN | DIFFERENTIATION | DATABASE | RNA | SEQUENCE | GENETICS & HEREDITY
Journal Article
Methods in Molecular Biology, ISSN 1064-3745, 2018, Volume 1828, pp. 439 - 454
Spinal muscular atrophy (SMA), the most common gentic cause of infantile death caused by mutations in the SMN1 gene, presents a unique case in the field of... 
Splice modulation therapy | Locked nucleic acids (LNA) | Survival of motor neuron (SMN) | Exon inclusion | Antisense oligonucleotides | Antisense therapy | Phosphorodiamidate morpholino oligomers (PMO morpholinos) | Spinal muscular atrophy (SMA) | Werdnig–Hoffmann disease | Nusinersen (Spinraza) | Index Medicus
Journal Article
Methods in Molecular Biology, ISSN 1064-3745, 2018, Volume 1828, pp. 263 - 273
Journal Article
Methods in Molecular Biology, ISSN 1064-3745, 2018, Volume 1828, pp. 455 - 465
Journal Article
PLoS ONE, ISSN 1932-6203, 03/2015, Volume 10, Issue 3, pp. e0120058 - e0120058
Journal Article
Nature, ISSN 0028-0836, 10/2011, Volume 478, Issue 7367, pp. 127 - 131
Journal Article
Proceedings of the National Academy of Sciences of the United States of America, ISSN 0027-8424, 8/2012, Volume 109, Issue 34, pp. 13763 - 13768
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
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
Journal Article
Journal Article