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Recent advancements in exon-skipping therapies using antisense oligonucleotides and genome editing for the treatment of various muscular dystrophies.
- Published in:
- Expert Reviews in Molecular Medicine, 2019, v. 21, p. 1, doi. 10.1017/erm.2019.5
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- Article
Genetic Approaches for the Treatment of Giant Axonal Neuropathy.
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- Journal of Personalized Medicine, 2023, v. 13, n. 1, p. 91, doi. 10.3390/jpm13010091
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- Publication type:
- Article
A Genotype-Phenotype Correlation Study of Exon Skip-Equivalent In-Frame Deletions and Exon Skip-Amenable Out-of-Frame Deletions across the DMD Gene to Simulate the Effects of Exon-Skipping Therapies: A Meta-Analysis.
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- Journal of Personalized Medicine, 2021, v. 11, n. 1, p. 46, doi. 10.3390/jpm11010046
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- Article
Genotype–Phenotype Correlations in Duchenne and Becker Muscular Dystrophy Patients from the Canadian Neuromuscular Disease Registry.
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- Journal of Personalized Medicine, 2020, v. 10, n. 4, p. 241, doi. 10.3390/jpm10040241
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- Article
Molecular Diagnosis and Novel Therapies for Neuromuscular Diseases.
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- 2020
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- Publication type:
- Editorial
Advances in Genetic Characterization and Genotype–Phenotype Correlation of Duchenne and Becker Muscular Dystrophy in the Personalized Medicine Era.
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- Journal of Personalized Medicine, 2020, v. 10, n. 3, p. 111, doi. 10.3390/jpm10030111
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- Publication type:
- Article
Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: Current Status of Therapeutic Approaches.
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- Journal of Personalized Medicine, 2019, v. 9, n. 1, p. 1, doi. 10.3390/jpm9010001
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- Publication type:
- Article
Multiple Exon Skipping in the Duchenne Muscular Dystrophy Hot Spots: Prospects and Challenges.
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- Journal of Personalized Medicine, 2018, v. 8, n. 4, p. 41, doi. 10.3390/jpm8040041
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- Article
Applications of CRISPR/Cas9 for the Treatment of Duchenne Muscular Dystrophy.
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- Journal of Personalized Medicine, 2018, v. 8, n. 4, p. 38, doi. 10.3390/jpm8040038
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- Publication type:
- Article
Immortalized Muscle Cell Model to Test the Exon Skipping Efficacy for Duchenne Muscular Dystrophy.
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- Journal of Personalized Medicine, 2017, v. 7, n. 4, p. 13, doi. 10.3390/jpm7040013
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- Publication type:
- Article
Antisense Therapy in Neurology.
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- Journal of Personalized Medicine, 2013, v. 3, n. 3, p. 144, doi. 10.3390/jpm3030144
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- Publication type:
- Article
Ensemble-Learning and Feature Selection Techniques for Enhanced Antisense Oligonucleotide Efficacy Prediction in Exon Skipping.
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- Pharmaceutics, 2023, v. 15, n. 7, p. 1808, doi. 10.3390/pharmaceutics15071808
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- Publication type:
- Article
Enhancing the Effectiveness of Oligonucleotide Therapeutics Using Cell-Penetrating Peptide Conjugation, Chemical Modification, and Carrier-Based Delivery Strategies.
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- Pharmaceutics, 2023, v. 15, n. 4, p. 1130, doi. 10.3390/pharmaceutics15041130
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- Publication type:
- Article
Recent Trends in Antisense Therapies for Duchenne Muscular Dystrophy.
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- Pharmaceutics, 2023, v. 15, n. 3, p. 778, doi. 10.3390/pharmaceutics15030778
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- Publication type:
- Article
Assessing the Role of Aquaporin 4 in Skeletal Muscle Function.
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- International Journal of Molecular Sciences, 2023, v. 24, n. 2, p. 1489, doi. 10.3390/ijms24021489
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- Publication type:
- Article
CRISPR Therapeutics for Duchenne Muscular Dystrophy.
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- International Journal of Molecular Sciences, 2022, v. 23, n. 3, p. 1832, doi. 10.3390/ijms23031832
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- Publication type:
- Article
A Dystrophin Exon-52 Deleted Miniature Pig Model of Duchenne Muscular Dystrophy and Evaluation of Exon Skipping.
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- International Journal of Molecular Sciences, 2021, v. 22, n. 23, p. 13065, doi. 10.3390/ijms222313065
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- Article
Natural History of a Mouse Model Overexpressing the Dp71 Dystrophin Isoform.
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- International Journal of Molecular Sciences, 2021, v. 22, n. 23, p. 12617, doi. 10.3390/ijms222312617
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- Article
Membrane Repair Deficit in Facioscapulohumeral Muscular Dystrophy.
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- International Journal of Molecular Sciences, 2020, v. 21, n. 15, p. 5575, doi. 10.3390/ijms21155575
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- Publication type:
- Article
Genome Editing for the Understanding and Treatment of Inherited Cardiomyopathies.
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- International Journal of Molecular Sciences, 2020, v. 21, n. 3, p. 733, doi. 10.3390/ijms21030733
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- Publication type:
- Article
DUX4 Signalling in the Pathogenesis of Facioscapulohumeral Muscular Dystrophy.
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- International Journal of Molecular Sciences, 2020, v. 21, n. 3, p. 729, doi. 10.3390/ijms21030729
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- Publication type:
- Article
Muscular Dystrophy: Disease Mechanisms and Therapies.
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- BioMed Research International, 2015, v. 2015, p. 1, doi. 10.1155/2015/456348
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- Publication type:
- Article
Cardiac therapies for Duchenne muscular dystrophy.
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- Therapeutic Advances in Neurological Disorders, 2023, p. 1, doi. 10.1177/17562864231182934
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- Publication type:
- Article
Cardiac therapies for Duchenne muscular dystrophy.
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- Therapeutic Advances in Neurological Disorders, 2023, v. 16, p. 1, doi. 10.1177/17562864231182934
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- Publication type:
- Article
α-Syntrophin-deficient skeletal muscle exhibits hypertrophy and aberrant formation of neuromuscular junctions during regeneration.
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- Journal of Cell Biology, 2002, v. 158, n. 6, p. 1097, doi. 10.1083/jcb.200204076
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- Article
Impaired regenerative capacity and lower revertant fibre expansion in dystrophin-deficient mdx muscles on DBA/2 background.
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- Scientific Reports, 2016, p. 38371, doi. 10.1038/srep38371
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- Publication type:
- Article
Genetic Approaches for the Treatment of Facioscapulohumeral Muscular Dystrophy.
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- Frontiers in Pharmacology, 2021, v. 11, p. N.PAG, doi. 10.3389/fphar.2021.642858
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- Publication type:
- Article
In Silico Screening Based on Predictive Algorithms as a Design Tool for Exon Skipping Oligonucleotides in Duchenne Muscular Dystrophy.
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- PLoS ONE, 2015, v. 10, n. 3, p. 1, doi. 10.1371/journal.pone.0120058
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- Publication type:
- Article
Mutation Types and Aging Differently Affect Revertant Fiber Expansion in Dystrophic <i>Mdx</i> and <i>Mdx52</i> Mice.
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- PLoS ONE, 2013, v. 8, n. 7, p. 1, doi. 10.1371/journal.pone.0069194
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- Publication type:
- Article
Antisense PMO Found in Dystrophic Dog Model Was Effective in Cells from Exon 7-Deleted DMD Patient.
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- PLoS ONE, 2010, v. 5, n. 8, p. 1, doi. 10.1371/journal.pone.0012239
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- Publication type:
- Article
α1-Syntrophin-deficient mice exhibit impaired muscle force recovery after osmotic shock.
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- Muscle & Nerve, 2014, v. 49, n. 5, p. 728, doi. 10.1002/mus.23990
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- Article
Cardio‐respiratory and phenotypic rescue of dystrophin/utrophin‐deficient mice by combination therapy.
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- EMBO Reports, 2022, v. 23, n. 6, p. 1, doi. 10.15252/embr.202153955
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- Article
Nonmechanical Roles of Dystrophin and Associated Proteins in Exercise, Neuromuscular Junctions, and Brains.
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- Brain Sciences (2076-3425), 2015, v. 5, n. 3, p. 275, doi. 10.3390/brainsci5030275
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- Article
The Dysferlinopathies Conundrum: Clinical Spectra, Disease Mechanism and Genetic Approaches for Treatments.
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- Biomolecules (2218-273X), 2024, v. 14, n. 3, p. 256, doi. 10.3390/biom14030256
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- Publication type:
- Article
Potential of Cell-Penetrating Peptide-Conjugated Antisense Oligonucleotides for the Treatment of SMA.
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- Molecules, 2024, v. 29, n. 11, p. 2658, doi. 10.3390/molecules29112658
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- Publication type:
- Article
Morpholino Treatment Improves Muscle Function and Pathology of Pitx1 Transgenic Mice.
- Published in:
- Molecular Therapy, 2014, v. 22, n. 2, p. 390, doi. 10.1038/mt.2013.263
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- Publication type:
- Article
Pharmacological Profile of Viltolarsen for the Treatment of Duchenne Muscular Dystrophy: A Japanese Experience.
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- Clinical Pharmacology, 2021, v. 13, p. 235, doi. 10.2147/CPAA.S288842
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- Publication type:
- Article
Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs.
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- Annals of Neurology, 2009, v. 65, n. 6, p. 667, doi. 10.1002/ana.21627
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- Publication type:
- Article
Muscle dysfunction caused by loss of Magel2 in a mouse model of Prader-Willi and Schaaf-Yang syndromes.
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- Human Molecular Genetics, 2016, v. 25, n. 17, p. 3798, doi. 10.1093/hmg/ddw225
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- Publication type:
- Article
Highly efficient in vivo delivery of PMO into regenerating myotubes and rescue in laminin-α2 chain-null congenital muscular dystrophy mice.
- Published in:
- Human Molecular Genetics, 2013, v. 22, n. 24, p. 4914, doi. 10.1093/hmg/ddt341
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- Publication type:
- Article
Eteplirsen in the treatment of Duchenne muscular dystrophy.
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- Drug Design, Development & Therapy, 2017, v. 11, p. 533, doi. 10.2147/dddt.s97635
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- Publication type:
- Article
Pharmacology and toxicology of eteplirsen and SRP-5051 for DMD exon 51 skipping: an update.
- Published in:
- Archives of Toxicology, 2022, v. 96, n. 1, p. 1, doi. 10.1007/s00204-021-03184-z
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- Publication type:
- Article
Pathogenic exon-trapping by SVA retrotransposon and rescue in Fukuyama muscular dystrophy.
- Published in:
- Nature, 2011, v. 478, n. 7367, p. 127, doi. 10.1038/nature10456
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- Publication type:
- Article
The Status of Exon Skipping as a Therapeutic Approach to Duchenne Muscular Dystrophy.
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- Molecular Therapy, 2011, v. 19, n. 1, p. 9, doi. 10.1038/mt.2010.219
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- Publication type:
- Article
In-frame Dystrophin Following Exon 51-Skipping Improves Muscle Pathology and Function in the Exon 52-Deficient mdx Mouse.
- Published in:
- Molecular Therapy, 2010, v. 18, n. 11, p. 1995, doi. 10.1038/mt.2010.186
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- Publication type:
- Article
Skipping Multiple Exons to Treat DMD--Promises and Challenges.
- Published in:
- Biomedicines, 2018, v. 6, n. 1, p. 1, doi. 10.3390/biomedicines6010001
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- Publication type:
- Article
Enhancing Antisense Oligonucleotide-Based Therapeutic Delivery with DG9, a Versatile Cell-Penetrating Peptide.
- Published in:
- Cells (2073-4409), 2023, v. 12, n. 19, p. 2395, doi. 10.3390/cells12192395
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- Publication type:
- Article
Restoring SMN Expression: An Overview of the Therapeutic Developments for the Treatment of Spinal Muscular Atrophy.
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- Cells (2073-4409), 2022, v. 11, n. 3, p. 417, doi. 10.3390/cells11030417
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- Publication type:
- Article
eSkip-Finder: a machine learning-based web application and database to identify the optimal sequences of antisense oligonucleotides for exon skipping.
- Published in:
- Nucleic Acids Research, 2021, v. 49, n. W1, p. W193, doi. 10.1093/nar/gkab442
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- Publication type:
- Article
Current understanding and treatment of cardiac and skeletal muscle pathology in laminin-α2 chain-deficient congenital muscular dystrophy.
- Published in:
- Application of Clinical Genetics, 2019, v. 12, p. 113, doi. 10.2147/TACG.S187481
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- Publication type:
- Article