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FEBS Letters, ISSN 0014-5793, 02/2019, Volume 593, Issue 3, pp. 253 - 276
Journal Article
07/2010
Duchenne muscular dystrophy (DMD), the most common and serious form of childhood muscle wasting is generally caused by protein-truncating mutations in the... 
Becker muscular dystrophy | exon skipping | Duchenne muscular dystrophy | personalized genetic medicines | clinical trials | morpholino oligomer
Web Resource
F1000Research, ISSN 2046-1402, 05/2019, Volume 8, p. 710
Recent approvals of oligonucleotide analogue drugs to alter gene expression have been welcomed by patient communities but not universally supported. These... 
Antisense oligonucleotide | Alternative splicing | Exon selection | Drugs | Disease | Transcription | Splicing | Oligonucleotides | Clinical trials | Antisense oligonucleotides | FDA approval | Gene expression | Patients | Monomers | Muscular dystrophy | Design | Nuclease | Mutation | Sulfur | Deoxyribonucleic acid--DNA
Journal Article
Molecular Therapy - Nucleic Acids, ISSN 2162-2531, 06/2018, Volume 11, Issue C, pp. 91 - 102
The severe childhood disease spinal muscular atrophy (SMA) arises from the homozygous loss of the survival motor neuron 1 gene ( ). A homologous gene... 
antisense oligonucleotide | intron retention | splice modification | MEDICINE, RESEARCH & EXPERIMENTAL | DNA-SEQUENCES | EXON | SPINAL MUSCULAR-ATROPHY | MESSENGER-RNA | SURVIVAL MOTOR-NEURON | UNTRANSLATED REGIONS | GENE-EXPRESSION | NEGATIVE POSTTRANSCRIPTIONAL REGULATION | UTR SEQUENCE MOTIF | SINGLE NUCLEOTIDE
Journal Article
Methods in Molecular Biology, ISSN 1064-3745, 2018, Volume 1828, pp. 219 - 228
Duplications of one or more dystrophin exons that disrupt the reading frame account for about 15% of all Duchenne cases, and like the more common genomic... 
Strand slippage | Duplications | Dystrophin | Multiple exon skipping | PCR artifact | Index Medicus
Journal Article
Lancet, The, ISSN 0140-6736, 2011, Volume 378, Issue 9791, pp. 595 - 605
Journal Article
Lancet Neurology, The, ISSN 1474-4422, 2009, Volume 8, Issue 10, pp. 918 - 928
Journal Article
2013, Springerbriefs in biochemistry and molecular biology, ISBN 3034806795
There is now compelling evidence that the complexity of higher organisms correlates with the relative amount of non-coding RNA rather than the number of... 
Eukaryotic Cells | genetics | Gene Expression Regulation | Genes, Regulator | Untranslated Regions | Non-coding RNA
Web Resource
FEBS letters, 12/2018
The Hippo pathway has emerged as a major eukaryotic signalling pathway and is increasingly the subject of intense interest, as are the key effectors of... 
Journal Article
Journal of Gene Medicine, ISSN 1099-498X, 2009, Volume 11, Issue 1, pp. 46 - 56
  Background Duchenne muscular dystrophy (DMD), a severe neuromuscular disorder, is caused by protein-truncating mutations in the dystrophin gene. Absence of... 
Antisense oligomer | mouse | Morpholino | B6Ros.Cg-Dmd | Duchenne muscular dystrophy | Exon skipping
Journal Article
2013, 2013, SpringerBriefs in Biochemistry and Molecular Biology, ISBN 9783034806787, 63
There is now compelling evidence that the complexity of higher organisms correlates with the relative amount of non-coding RNA rather than the number of... 
Biology, life sciences | Gene expression | Genetic regulation | Life Sciences | Gene Function | Nucleic Acid Chemistry | Animal Genetics and Genomics | Plant Genetics & Genomics
eBook
PLoS ONE, ISSN 1932-6203, 2011, Volume 6, Issue 10, pp. e26820 - e26820
Background: Facioscapulohumeral muscular dystrophy (FSHD) is linked to deletions in 4q35 within the D4Z4 repeat array in which we identified the double... 
FACIOSCAPULOHUMERAL MUSCULAR-DYSTROPHY | SKELETAL-MUSCLE ATROPHY | PROTEIN | MESSENGER-RNA | GENE | THYROID-HORMONE | D4Z4 | MULTIDISCIPLINARY SCIENCES | IN-VIVO | MU-CRYSTALLIN | LOCUS | RNA Interference - drug effects | Homeodomain Proteins - metabolism | Humans | Muscle Fibers, Skeletal - drug effects | Muscle Fibers, Skeletal - metabolism | RNA, Messenger - metabolism | Tripartite Motif Proteins | Transfection | RNA Splicing - genetics | Muscle Proteins - metabolism | Muscular Dystrophy, Facioscapulohumeral - metabolism | Biomarkers - metabolism | RNA Splicing - drug effects | Oligonucleotides, Antisense - pharmacology | Muscular Atrophy - metabolism | Muscular Atrophy - pathology | RNA, Messenger - genetics | Cells, Cultured | Ubiquitin-Protein Ligases - metabolism | SKP Cullin F-Box Protein Ligases - metabolism | Down-Regulation - drug effects | Homeodomain Proteins - genetics | Gene Expression Regulation - drug effects | Phenotype | Animals | Models, Biological | Muscle Fibers, Skeletal - pathology | Muscular Dystrophy, Facioscapulohumeral - pathology | Mice | RNA, Small Interfering - metabolism | Ubiquitin | Infection | Messenger RNA | Ligases | Genes | Genetic aspects | Tumor proteins | Health aspects | Oxidative stress | Deregulation | Senescence | Transcription factors | Laboratories | p53 Protein | Oligonucleotides | Thyroid gland | Kinases | Muscular dystrophy | Homeobox | Proteins | Atrophy | Rodents | Aging | Telomerase | Antisense RNA | Cultures | Antisense oligonucleotides | Hearing impairment | Myoblasts | Studies | Crystallin | Polyadenylation | Reagents | Mutation | Dystrophy | Molecular biology | Index Medicus
Journal Article
Journal Article