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Molecular Therapy - Nucleic Acids, ISSN 2162-2531, 2015, Volume 4, Issue 2, pp. e225 - e225
Antisense-mediated exon skipping, which can restore the reading frame, is a most promising therapeutic approach for Duchenne muscular dystrophy. Remaining... 
multiexon skipping | antisense therapeutics | mdx52 mouse | dystrophin | Vivo-Morpholinos | Duchenne muscular dystrophy | Antisense therapeutics | Dystrophin | Multiexon skipping | Mmdx52 mouse | MUSCLE PATHOLOGY | MEDICINE, RESEARCH & EXPERIMENTAL | CARDIAC-MUSCLE | RESTORATION | DETERMINES | THERAPY | RESCUE | IMPROVEMENT | DUCHENNE MUSCULAR-DYSTROPHY | SKELETAL | EXPRESSION | Original
Journal Article
MOLECULAR THERAPY, ISSN 1525-0016, 04/2015, Volume 23, Issue 4, pp. 707 - 716
Despite the medical, social, and economic impact of obesity, only a few therapeutic options, focused largely on reducing caloric intake, are currently... 
SENSITIVE POTASSIUM CHANNELS | MEDICINE, RESEARCH & EXPERIMENTAL | ENERGY | FATIGUE | HEART-FAILURE | SOLEUS MUSCLE | MDX MICE | OBESITY | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | VIVO-MORPHOLINOS | FIBER DAMAGE | GENETICS & HEREDITY | FORCE DEVELOPMENT
Journal Article
Methods in Molecular Biology, ISSN 1064-3745, 2018, Volume 1828, pp. 275 - 292
Exon-skipping therapy is an emerging approach that uses synthetic DNA-like molecules called antisense oligonucleotides (ASOs) to splice out frame-disrupting... 
mdx | mdx52 | Exon skipping | Duchenne muscular dystrophy (DMD) | Eteplirsen (Exondys 51) | Vivo-morpholinos (vPMOs) | Dystrophin | Multiexon skipping | Becker muscular dystrophy (BMD) | Phosphorodiamidate morpholino oligomer (PMO) | Index Medicus
Journal Article
RSC Advances, ISSN 2046-2069, 07/2015, Volume 5, Issue 80, pp. 65245 - 65254
A convenient approach to the delivery of uncharged peptide nucleic acids (PNA) or phosphorodiamidate morpholino (PMO) oligomers in mammalian cells is presented... 
MDX MOUSE | EXON | PNA | GENE DELIVERY | VIVO-MORPHOLINOS | ANTISENSE MORPHOLINO OLIGOMERS | DUCHENNE MUSCULAR-DYSTROPHY | CELLULAR DELIVERY | EFFICIENT SPLICING CORRECTION | CHEMISTRY, MULTIDISCIPLINARY | PENETRATING PEPTIDES | Oligomers | Uptakes | Biotechnology | Splicing | Deoxyribonucleic acid | Nucleic acids | Mammals | Recognition
Journal Article
Journal Article
PLoS ONE, ISSN 1932-6203, 01/2015, Volume 10, Issue 1, pp. e0114932 - e0114932
Traditional gene targeting methods in mice are complex and time consuming, especially when conditional deletion methods are required. Here, we describe a novel... 
MULTIDISCIPLINARY SCIENCES | VIVO-MORPHOLINOS | IN-VIVO | SEX DETERMINATION | BRANCHING-MORPHOGENESIS | KIDNEY DEVELOPMENT | PREMATURE OVARIAN FAILURE | TESTIS DEVELOPMENT | SOX9 | CELL FATE | EXPRESSION | Gonads - embryology | Gene Targeting - methods | Testis - metabolism | Chymotrypsin - metabolism | Embryo, Mammalian | Male | Testis - embryology | Kruppel-Like Transcription Factors - metabolism | Female | Chymotrypsin - genetics | SOX9 Transcription Factor - metabolism | Morpholinos - administration & dosage | Embryonic Development - genetics | Pancreas - metabolism | Gene Knockout Techniques | Pancreas - embryology | ADAM Proteins - metabolism | Gonads - metabolism | Injections | Animals | Organogenesis - genetics | Adaptor Proteins, Signal Transducing - genetics | Mice | Zinc Finger Protein GLI1 | ADAM Proteins - genetics | Adaptor Proteins, Signal Transducing - metabolism | Kruppel-Like Transcription Factors - genetics | SOX9 Transcription Factor - genetics | Pregnancy | Embryonic development | Analysis | Genes | Heart | Sox9 protein | Genomics | Fetuses | Oligonucleotides | Antisense oligonucleotides | Genomes | Gestation | Cell differentiation | Gene expression | Medical screening | Embryos | Organogenesis | Defects | Gene targeting | Clonal deletion | Sex determination | Animal tissues | Genetic analysis | Deletion | Genetic engineering | Pancreas | Gonads | Methods | Index Medicus
Journal Article
Journal Article
Human Mutation, ISSN 1059-7794, 01/2012, Volume 33, Issue 1, pp. 198 - 208
A recent challenge for investigators studying the progressive neurological disease ataxia-telangiectasia (A-T) is to identify mutations whose effects might be... 
ATM | Large genomic deletions | Ataxia-telangiectasia | Functional analysis of DNA variants | Mutationtargeted therapy | Japanese ATM mutation | POPULATION | ACTIVATION | DNA-DAMAGE | HAPLOTYPES | large genomic deletions | 55-PERCENT | ataxia-telangiectasia | functional analysis of DNA variants | GENE | EXONIC SPLICING ENHANCERS | FAMILIES | VIVO-MORPHOLINOS | GENETICS & HEREDITY | mutation-targeted therapy | AUTOPHOSPHORYLATION | Sequence Deletion | Frameshift Mutation | Oligodeoxyribonucleotides, Antisense - pharmacology | Exons | Humans | Morpholinos - therapeutic use | Molecular Sequence Data | Molecular Targeted Therapy | RNA Splicing | DNA-Binding Proteins - agonists | T-Lymphocytes - metabolism | DNA Mutational Analysis | Gentamicins - pharmacology | T-Lymphocytes - drug effects | Base Sequence | Tumor Suppressor Proteins - genetics | Cell Cycle Proteins - genetics | Ataxia Telangiectasia - drug therapy | T-Lymphocytes - pathology | Cell Line | Gentamicins - therapeutic use | Protein-Serine-Threonine Kinases - genetics | Oligodeoxyribonucleotides, Antisense - therapeutic use | Ataxia Telangiectasia Mutated Proteins | Cell Cycle Proteins - agonists | Codon, Nonsense | DNA-Binding Proteins - genetics | Asian Continental Ancestry Group | Pedigree | Morpholinos - pharmacology | Ataxia Telangiectasia - genetics | Heterozygote | Aminoglycosides - pharmacology | Tumor Suppressor Proteins - agonists | Aminoglycosides - therapeutic use | Index Medicus
Journal Article
Journal of Visualized Experiments, ISSN 1940-087X, 05/2016, Volume 2016, Issue 111
Journal Article
ChemistrySelect, ISSN 2365-6549, 06/2017, Volume 2, Issue 18, pp. 5059 - 5067
Cationic guanidinium and phosphonium functionalized cytosine morpholino tetramer (G3) and trimer (P3) have been reported for the first time. They show... 
phosphonium group | guanidinium group | zebrafish | cationic morpholino | Gli1 | cellular transfection | antisense | Gli1 antisense | CELL-PENETRATING PEPTIDE | NUCLEIC-ACID | CHEMISTRY, MULTIDISCIPLINARY | DELIVERY | MOLECULAR TRANSPORTER | VIVO-MORPHOLINOS | DUCHENNE MUSCULAR-DYSTROPHY | ANTISENSE OLIGONUCLEOTIDES | GLI GENES | EFFICIENT | HEDGEHOG SIGNALING PATHWAY
Journal Article
Frontiers in Microbiology, ISSN 1664-302X, 04/2018, Volume 9, pp. 750 - 750
Phosphorodiamidate morpholino oligomers (PMO) are short single-stranded DNA analogs that are built upon a backbone of morpholine rings connected by... 
Morpholino oligomers | Antiviral compound | Antisense | PMO | PPMO | antiviral compound | SARCOMA-ASSOCIATED HERPESVIRUS | MICROBIOLOGY | morpholino oligomers | CELL-CULTURES | PEPTIDE | INHIBITION | VIVO-MORPHOLINOS | IN-VIVO | STERIC-BLOCK OLIGONUCLEOTIDES | RIBOSOME ENTRY SITE | PHOSPHOROTHIOATE OLIGONUCLEOTIDE | antisense | EBOLA-VIRUS | Oligomers | Health aspects
Journal Article