Found: 14
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Microdystrophin Gene Addition Significantly Improves Muscle Functionality and Diaphragm Muscle Histopathology in a Fibrotic Mouse Model of Duchenne Muscular Dystrophy.
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- International Journal of Molecular Sciences, 2023, v. 24, n. 9, p. 8174, doi. 10.3390/ijms24098174
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- Article
Combination Antisense Treatment for Destructive Exon Skipping of Myostatin and Open Reading Frame Rescue of Dystrophin in Neonatal mdx Mice.
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- Molecular Therapy, 2015, v. 23, n. 8, p. 1341, doi. 10.1038/mt.2015.88
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- Article
Systemic antisense therapeutics inhibiting DUX4 expression ameliorates FSHD-like pathology in an FSHD mouse model.
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- Human Molecular Genetics, 2021, v. 30, n. 15, p. 1398, doi. 10.1093/hmg/ddab136
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- Article
Established PABPN1 intranuclear inclusions in OPMD muscle can be efficiently reversed by AAV-mediated knockdown and replacement of mutant expanded PABPN1.
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- Human Molecular Genetics, 2019, v. 28, n. 19, p. 3301, doi. 10.1093/hmg/ddz167
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- Article
Functional muscle recovery following dystrophin and myostatin exon splice modulation in aged mdx mice.
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- Human Molecular Genetics, 2019, v. 28, n. 18, p. 3091, doi. 10.1093/hmg/ddz125
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- Article
Pharmacological modulation of the ER stress response ameliorates oculopharyngeal muscular dystrophy.
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- Human Molecular Genetics, 2019, v. 28, n. 10, p. 1694, doi. 10.1093/hmg/ddz007
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- Article
Expression of the Pro-Fibrotic Marker Periostin in a Mouse Model of Duchenne Muscular Dystrophy.
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- Biomedicines, 2024, v. 12, n. 1, p. 216, doi. 10.3390/biomedicines12010216
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- Article
An Induced Pluripotent Stem Cell-Derived Human Blood–Brain Barrier (BBB) Model to Test the Crossing by Adeno-Associated Virus (AAV) Vectors and Antisense Oligonucleotides.
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- Biomedicines, 2023, v. 11, n. 10, p. 2700, doi. 10.3390/biomedicines11102700
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- Article
Long-Term Systemic Treatment of a Mouse Model Displaying Chronic FSHD-like Pathology with Antisense Therapeutics That Inhibit DUX4 Expression.
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- Biomedicines, 2022, v. 10, n. 7, p. N.PAG, doi. 10.3390/biomedicines10071623
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- Article
Targeting TGFβ Signaling to Address Fibrosis Using Antisense Oligonucleotides.
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- Biomedicines, 2018, v. 6, n. 3, p. 74, doi. 10.3390/biomedicines6030074
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- Article
G-quadruplex ligands mediate downregulation of DUX4 expression.
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- Nucleic Acids Research, 2020, v. 48, n. 8, p. 4179, doi. 10.1093/nar/gkaa146
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- Article
Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy.
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- Journal of Cachexia, Sarcopenia & Muscle, 2020, v. 11, n. 3, p. 768, doi. 10.1002/jcsm.12542
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- Article
Inhibition of myostatin improves muscle atrophy in oculopharyngeal muscular dystrophy (OPMD).
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- Journal of Cachexia, Sarcopenia & Muscle, 2019, v. 10, n. 5, p. 1016, doi. 10.1002/jcsm.12438
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- Article
Targeted Antisense Oligonucleotide-Mediated Skipping of Murine Postn Exon 17 Partially Addresses Fibrosis in D2. mdx Mice.
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- International Journal of Molecular Sciences, 2024, v. 25, n. 11, p. 6113, doi. 10.3390/ijms25116113
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- Article