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duchenne muscular dystrophy (2) 2
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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
Journal of Neuromuscular Diseases, ISSN 2214-3599, 2016, Volume 3, Issue 1, pp. 29 - 48
Duchenne muscular dystrophy (DMD) is an X-linked genetic disorder characterized by progressive muscle degeneration. Mutations in the DMD gene result in the... 
DBA/2-mdx | mdx | exon skipping | C57BL/6-mdx | mdx52, hDMD | dko | Duchenne muscular dystrophy (DMD) | Cmah-mdx | DMD-null
Journal Article
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