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The administration of antisense oligonucleotide golodirsen reduces pathological regeneration in patients with Duchenne muscular dystrophy.
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- Acta Neuropathologica Communications, 2021, v. 9, n. 1, p. 1, doi. 10.1186/s40478-020-01106-1
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- Article
Restoration of Normal NF1 Function with Antisense Morpholino Treatment of Recurrent Pathogenic Patient-Specific Variant c.1466A>G; p.Y489C.
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- Journal of Personalized Medicine, 2021, v. 11, n. 12, p. 1320, doi. 10.3390/jpm11121320
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- Article
Inhibition of Myostatin Reduces Collagen Deposition in a Mouse Model of Oculopharyngeal Muscular Dystrophy (OPMD) With Established Disease.
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- Frontiers in Physiology, 2020, v. 11, p. 1, doi. 10.3389/fphys.2020.00184
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- Article
Reversible immortalisation enables genetic correction of human muscle progenitors and engineering of next‐generation human artificial chromosomes for Duchenne muscular dystrophy.
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- EMBO Molecular Medicine, 2018, v. 10, n. 2, p. 254, doi. 10.15252/emmm.201607284
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- Article
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
Novel Metabolomic Approach for Identifying Pathology-Specific Biomarkers in Rare Diseases: A Case Study in Oculopharyngeal Muscular Dystrophy (OPMD).
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- Metabolites (2218-1989), 2023, v. 13, n. 6, p. 769, doi. 10.3390/metabo13060769
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- Article
Nuclear poly(A)-binding protein aggregates misplace a pre-mRNA outside of SC35 speckle causing its abnormal splicing.
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- Nucleic Acids Research, 2016, v. 44, n. 22, p. 10929, doi. 10.1093/nar/gkw703
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- Article
RAC1B modulates intestinal tumourigenesis via modulation of WNT and EGFR signalling pathways.
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- Nature Communications, 2021, v. 12, n. 1, p. 1, doi. 10.1038/s41467-021-22531-3
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- Article
Dp412e: a novel human embryonic dystrophin isoform induced by BMP4 in early differentiated cells.
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- Skeletal Muscle, 2015, v. 5, p. 1, doi. 10.1186/s13395-015-0062-6
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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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- Publication type:
- 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
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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- Publication type:
- Article
Antisense targeting of 30 end elements involved in DUX4 mRNA processing is an efficient therapeutic strategy for facioscapulohumeral dystrophy: a new gene-silencing approach.
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- Human Molecular Genetics, 2016, v. 25, n. 8, p. 1468, doi. 10.1093/hmg/ddw015
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- Article
β-catenin as a potential key target for tumor suppression.
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- International Journal of Cancer, 2011, v. 129, n. 7, p. 1541, doi. 10.1002/ijc.26102
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- Publication type:
- Article
Antisense-induced Myostatin Exon Skipping Leads to Muscle Hypertrophy in Mice Following Octa guanidine Morpholino Oligomer Treatment.
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- Molecular Therapy, 2011, v. 19, n. 1, p. 159, doi. 10.1038/mt.2010.212
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- Publication type:
- Article
Design of Phosphorodiamidate Morpholino Oligomers (PMOs) for the Induction of Exon Skipping of the Human DMD Gene.
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- Molecular Therapy, 2009, v. 17, n. 3, p. 554, doi. 10.1038/mt.2008.287
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- Publication type:
- 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
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
Editorial of the Special Issue: Antisense Therapies.
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- 2018
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- Publication type:
- Editorial
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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- Publication type:
- 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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- Publication type:
- Article
Novel Cationic Carotenoid Lipids as Delivery Vectors of Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy.
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- Molecules, 2012, v. 17, n. 2, p. 1138, doi. 10.3390/molecules17021138
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- Publication type:
- 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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- Publication type:
- Article
A multicenter comparison of quantification methods for antisense oligonucleotide-induced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures.
- Published in:
- PLoS ONE, 2018, v. 13, n. 10, p. 1, doi. 10.1371/journal.pone.0204485
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- Publication type:
- 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