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Proceedings of the National Academy of Sciences of the United States of America, ISSN 0027-8424, 9/2016, Volume 113, Issue 39, pp. 10962 - 10967
Journal Article
Molecular Therapy - Nucleic Acids, ISSN 2162-2531, 2012, Volume 1, Issue 8, pp. e38 - e38
Antisense oligonucleotides (AOs) are currently the most promising therapeutic intervention for Duchenne muscular dystrophy (DMD). AOs modulate dystrophin... 
antisense oligonucleotide | cardiac muscle | PMO internalizing peptide | Duchenne muscular dystrophy | phosphorodiamidate morpholino oligomer | Phosphorodiamidate morpholino oligomer | Cardiac muscle | Antisense oligonucleotide | MEDICINE, RESEARCH & EXPERIMENTAL | Original
Journal Article
Current Gene Therapy, ISSN 1566-5232, 06/2012, Volume 12, Issue 3, pp. 161 - 178
The hydrophobic plasma membrane constitutes an indispensable barrier for cells, allowing influx of essential molecules while preventing access to other... 
Alternative splicing | Gene regulation | Macromolecules | Double-stranded RNA | Oligonucleotides | Antisense | Drug delivery | siRNA | Hydrophobicity | Drug development | Gene silencing | Duchenne's muscular dystrophy | Plasma membranes | spinal muscular atrophy | Gene therapy
Journal Article
Journal Article
Scientific Reports, ISSN 2045-2322, 03/2015, Volume 5, Issue 1, p. 8986
Journal Article
Scientific Reports, ISSN 2045-2322, 06/2015, Volume 5, Issue 1, p. 11632
Duchenne muscular dystrophy (DMD) is caused by absence of the integral structural protein, dystrophin, which renders muscle fibres susceptible to injury and... 
HEART | FIBROSIS | SYSTEMIC DELIVERY | GENE | MORPHOLINO OLIGOMERS | MULTIDISCIPLINARY SCIENCES | CARDIOMYOPATHY | MUSCLE | ANTISENSE OLIGONUCLEOTIDES | EXPRESSION | MDX MICE | Gene Expression - drug effects | Humans | NADPH Oxidases - metabolism | Male | Muscle, Skeletal - metabolism | Muscular Dystrophy, Duchenne - physiopathology | Muscle, Skeletal - drug effects | Mice, Inbred mdx | NADPH Oxidases - genetics | Diaphragm - metabolism | Dystrophin - metabolism | Diaphragm - physiopathology | Disease Models, Animal | Heart - physiopathology | Amino Acid Sequence | Protein Precursors - genetics | Peptides - chemistry | Mice, Inbred C57BL | NADPH Oxidase 4 | Diaphragm - diagnostic imaging | Physical Conditioning, Animal - physiology | Reverse Transcriptase Polymerase Chain Reaction | Blotting, Western | Morpholinos - chemistry | Peptides - pharmacology | Protein Precursors - metabolism | Radiography | Magnetic Resonance Imaging | Animals | Dystrophin - genetics | Natriuretic Peptide, C-Type - metabolism | Muscle, Skeletal - physiopathology | Morpholinos - pharmacology | Natriuretic Peptide, C-Type - genetics | Muscular Dystrophy, Duchenne - genetics | Magnetic resonance imaging | Cardiac muscle | Rodents | Exon skipping | Duchenne's muscular dystrophy | Oligonucleotides | Degeneration | Diaphragm | Dystrophy | Dystrophin | Heart diseases | Muscular dystrophy
Journal Article
Current Gene Therapy, ISSN 1566-5232, 06/2012, Volume 12, Issue 3, pp. 161 - 178
Journal Article
Journal Article
Proceedings of the National Academy of Sciences of the United States of America, ISSN 0027-8424, 09/2016, Volume 113, Issue 39, p. 10962
  The development of antisense oligonucleotide therapy is an important advance in the identification of corrective therapy for neuromuscular diseases, such as... 
Musculoskeletal diseases | Proteins | Therapy | Peptides | Mutation | Gene expression
Journal Article
Proceedings of the National Academy of Sciences, USA, ISSN 0027-8424, 09/2016, Volume 113, Issue 39, pp. 10962 - 10962
The development of antisense oligonucleotide therapy is an important advance in the identification of corrective therapy for neuromuscular diseases, such as... 
Journal Article
nucleic acid therapeutics, ISSN 2159-3337, 04/2015, Volume 25, Issue 2, pp. 65 - 77
Journal Article
Trends in Genetics, ISSN 0168-9525, 2011, Volume 27, Issue 5, pp. 196 - 205
Journal Article
Journal Article
Methods in molecular biology (Clifton, N.J.), 2019, Volume 2036, p. 221
Antisense oligonucleotides (ASOs) are a widely used form of gene therapy, which is translatable to multiple disorders. A major obstacle for ASO efficacy is its... 
Journal Article
American Journal of Epidemiology, ISSN 0002-9262, 05/2013, Volume 177, Issue 10, p. 1074
  Congenital anomalies are a leading cause of infant mortality and are important contributors to subsequent morbidity. Studies suggest associations between... 
Congenital diseases | Carbon dioxide | Air pollution | Infant mortality | Chemical compounds | Morbidity
Journal Article