X
Search Filters
Format Format
Subjects Subjects
Subjects Subjects
X
Sort by Item Count (A-Z)
Filter by Count
index medicus (53) 53
restores dystrophin expression (51) 51
humans (49) 49
animals (47) 47
duchenne muscular-dystrophy (23) 23
medicine, research & experimental (22) 22
mice (22) 22
systemic delivery (22) 22
antisense oligonucleotides (21) 21
restore dystrophin (18) 18
biochemistry & molecular biology (17) 17
cell biology (16) 16
muscular dystrophy (16) 16
messenger-rna (15) 15
dystrophin (14) 14
genetics & heredity (14) 14
in-vivo (14) 14
muscular dystrophy, duchenne - therapy (14) 14
mutation (14) 14
stem cells (14) 14
genetic therapy (13) 13
mouse model (13) 13
muscular-dystrophy (13) 13
duchenne muscular dystrophy (12) 12
exon skipping (12) 12
genetic therapy - methods (12) 12
skeletal-muscle (12) 12
muscular dystrophy, duchenne - genetics (11) 11
neurosciences (11) 11
oligonucleotides, antisense - therapeutic use (11) 11
review (11) 11
satellite cells (11) 11
article (10) 10
gene therapy (10) 10
muscle (10) 10
oligonucleotides, antisense - genetics (10) 10
pathology (10) 10
pluripotent stem-cells (10) 10
proteins (10) 10
spinal muscular-atrophy (10) 10
biotechnology & applied microbiology (9) 9
exons - genetics (9) 9
gene-expression (9) 9
gene-therapy (9) 9
rna splicing (9) 9
care and treatment (8) 8
clinical neurology (8) 8
dystrophin - genetics (8) 8
exons (8) 8
expression (8) 8
gene expression (8) 8
mdx mice (8) 8
mdx mouse (8) 8
mice, inbred mdx (8) 8
musculoskeletal diseases (8) 8
oligonucleotides, antisense - pharmacology (8) 8
pharmacology & pharmacy (8) 8
rna, messenger - genetics (8) 8
rodents (8) 8
cell differentiation (7) 7
gene (7) 7
messenger rna (7) 7
molecular biology (7) 7
multidisciplinary sciences (7) 7
muscles (7) 7
research (7) 7
rna (7) 7
skeletal muscle (7) 7
stem cell transplantation (7) 7
transplantation (7) 7
alternative splicing (6) 6
analysis (6) 6
cells (6) 6
clinical trials (6) 6
disease models, animal (6) 6
dystrophy (6) 6
genetic aspects (6) 6
genetics (6) 6
health aspects (6) 6
life sciences (6) 6
molecular medicine (6) 6
mouse (6) 6
musculoskeletal system (6) 6
phenotype (6) 6
research article (6) 6
restoration (6) 6
antisense (5) 5
antisense oligonucleotide (5) 5
cell line (5) 5
congenital, hereditary, and neonatal diseases and abnormalities (5) 5
disease (5) 5
exon (5) 5
genes (5) 5
genetic diseases, inborn - therapy (5) 5
genetic disorders (5) 5
genetic engineering (5) 5
in-vitro (5) 5
medical research (5) 5
morpholino oligomers (5) 5
muscle, skeletal - physiology (5) 5
more...
Language Language
Publication Date Publication Date
Click on a bar to filter by decade
Slide to change publication date range


Molecular Therapy - Nucleic Acids, ISSN 2162-2531, 2015, Volume 4, Issue 10, pp. e255 - e255
Antisense oligonucleotide (AO)-mediated exon-skipping therapeutics shows great promise for Duchenne muscular dystrophy (DMD) patients. However, recent failure... 
dystrophin | antisense oligonucleotide | exon skipping | Duchenne muscular dystrophy | peptide nucleic acid | MEDICINE, RESEARCH & EXPERIMENTAL | UTROPHIN | RESTORATION | MODEL | DELIVERY | RESTORES | EXON | DNA | DUCHENNE MUSCULAR-DYSTROPHY | GENE-THERAPY | ANTISENSE OLIGONUCLEOTIDES | Original
Journal Article
Nucleic Acids Research, ISSN 0305-1048, 08/2016, Volume 44, Issue 14, pp. 6549 - 6563
Journal Article
Journal Article
Journal of Controlled Release, ISSN 0168-3659, 2009, Volume 134, Issue 2, pp. 132 - 140
Journal Article
Journal of Molecular and Cellular Cardiology, ISSN 0022-2828, 04/2019, Volume 129, pp. 272 - 280
Cardiovascular disease (CVD) remains the leading cause of death in the Western world. Despite advances in the prevention and in the management of CVD, the role... 
RNA | Epigenetics | Cardiovascular | RESTORES DYSTROPHIN EXPRESSION | METHYLATION | CARDIAC & CARDIOVASCULAR SYSTEMS | CELL BIOLOGY | OBESITY | MESSENGER-RNA | GENE | WRITERS | NUCLEAR-RNA | PROMOTES | SUBUNIT | Prevention | Epigenetic inheritance | Research institutes | Genetic transcription | Cardiovascular diseases | Methylation | Cells
Journal Article
Molecular Therapy - Nucleic Acids, ISSN 2162-2531, 06/2017, Volume 7, Issue C, pp. 11 - 19
Exonic duplications account for 10%–15% of all mutations in Duchenne muscular dystrophy (DMD), a severe hereditary neuromuscular disorder. We report a CRISPR... 
dystrophin | CRISPR/Cas9 | gene editing | duplication | lentivirus | MEDICINE, RESEARCH & EXPERIMENTAL | CELLS | RESTORES DYSTROPHIN EXPRESSION | GENE | MOUSE MODEL | MUSCULAR-DYSTROPHY | MUSCLE | DUCHENNE | MUTATIONS | REARRANGEMENTS | NUCLEASES | CRISPR | Myotubes | Transcription | Genomes | Ribonucleic acid--RNA | Gene deletion | Muscular dystrophy | gRNA | Myoblasts | Proteins | Reading | Clonal deletion | Duchenne's muscular dystrophy | Mutation | Dystrophy | Dystrophin | Cas9 | Original
Journal Article
American Journal of Kidney Diseases, ISSN 0272-6386, 06/2018, Volume 71, Issue 6, pp. 874 - 883
Journal Article