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Cardio‐respiratory and phenotypic rescue of dystrophin/utrophin‐deficient mice by combination therapy.
- Published in:
- EMBO Reports, 2022, v. 23, n. 6, p. 1, doi. 10.15252/embr.202153955
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- Article
Anchor peptide captures, targets, and loads exosomes of diverse origins for diagnostics and therapy.
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- Science Translational Medicine, 2018, v. 10, n. 444, p. 1, doi. 10.1126/scitranslmed.aat0195
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- Article
Effective Dystrophin Restoration by a Novel Muscle-Homing Peptide-Morpholino Conjugate in Dystrophin-Deficient mdx Mice.
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- Molecular Therapy, 2014, v. 22, n. 7, p. 1333, doi. 10.1038/mt.2014.63
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- Article
Pip5 Transduction Peptides Direct High Efficiency Oligonucleotide-mediated Dystrophin Exon Skipping in Heart and Phenotypic Correction in mdx Mice.
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- Molecular Therapy, 2011, v. 19, n. 7, p. 1295, doi. 10.1038/mt.2011.79
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- Publication type:
- Article
Functional Rescue of Dystrophin-deficient mdx Mice by a Chimeric Peptide-PMO.
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- Molecular Therapy, 2010, v. 18, n. 10, p. 1822, doi. 10.1038/mt.2010.151
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- Publication type:
- Article
Optimization of Peptide Nucleic Acid Antisense Oligonucleotides for Local and Systemic Dystrophin Splice Correction in the mdx Mouse.
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- Molecular Therapy, 2010, v. 18, n. 4, p. 819, doi. 10.1038/mt.2009.310
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- Publication type:
- Article
Effective Exon Skipping and Restoration of Dystrophin Expression by Peptide Nucleic Acid Antisense Oligonucleotides in mdx Mice.
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- Molecular Therapy, 2008, v. 16, n. 1, p. 38, doi. 10.1038/sj.mt.6300329
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- Article
788. Antisense Therapy for Duchenne Muscular Dystrophy: A Realistic Possibility
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- 2005
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- Abstract
Alarmin-painted exosomes elicit persistent antitumor immunity in large established tumors in mice.
- Published in:
- Nature Communications, 2020, v. 11, n. 1, p. 1, doi. 10.1038/s41467-020-15569-2
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- Article
Whole exome sequencing identified a novel DAG1 mutation in a patient with rare, mild and late age of onset muscular dystrophy‐dystroglycanopathy.
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- Journal of Cellular & Molecular Medicine, 2019, v. 23, n. 2, p. 811, doi. 10.1111/jcmm.13979
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- Article
Milk‐derived extracellular vesicles functionalized with anti‐tumour necrosis factor‐α nanobody and anti‐microbial peptide alleviate ulcerative colitis in mice.
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- Journal of Extracellular Vesicles, 2024, v. 13, n. 6, p. 1, doi. 10.1002/jev2.12462
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- Publication type:
- Article
Exosome-mediated delivery of an anti-angiogenic peptide inhibits pathological retinal angiogenesis.
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- Theranostics, 2021, v. 11, n. 11, p. 5107, doi. 10.7150/thno.54755
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- Article
Universal immunotherapeutic strategy for hepatocellular carcinoma with exosome vaccines that engage adaptive and innate immune responses.
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- Journal of Hematology & Oncology, 2022, v. 15, n. 1, p. 1, doi. 10.1186/s13045-022-01266-8
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- Publication type:
- Article
In vitro evaluation of novel antisense oligonucleotides is predictive of in vivo exon skipping activity for Duchenne muscular dystrophy.
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- Journal of Gene Medicine, 2010, v. 12, n. 4, p. 354, doi. 10.1002/jgm.1446
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- Publication type:
- Article
Diaphragm rescue alone prevents heart dysfunction in dystrophic mice.
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- Human Molecular Genetics, 2011, v. 20, n. 3, p. 413, doi. 10.1093/hmg/ddq477
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- Publication type:
- Article
A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice.
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- Human Molecular Genetics, 2009, v. 18, n. 22, p. 4405, doi. 10.1093/hmg/ddp395
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- Publication type:
- Article
Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function.
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- Human Molecular Genetics, 2008, v. 17, n. 24, p. 3909, doi. 10.1093/hmg/ddn293
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- Publication type:
- Article
Hexose enhances oligonucleotide delivery and exon skipping in dystrophin-deficient mdx mice.
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- Nature Communications, 2016, v. 7, n. 3, p. 10981, doi. 10.1038/ncomms10981
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- Publication type:
- Article
Wild-Type Mouse Models to Screen Antisense Oligonucleotides for Exon-Skipping Efficacy in Duchenne Muscular Dystrophy.
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- PLoS ONE, 2014, v. 9, n. 11, p. 1, doi. 10.1371/journal.pone.0111079
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- Publication type:
- Article
Effective Exon Skipping and Dystrophin Restoration by 2′-O-Methoxyethyl Antisense Oligonucleotide in Dystrophin-Deficient Mice.
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- PLoS ONE, 2013, v. 8, n. 4, p. 1, doi. 10.1371/journal.pone.0061584
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- Publication type:
- Article
Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology.
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- Nature Medicine, 2006, v. 12, n. 2, p. 175, doi. 10.1038/nm1345
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- Publication type:
- Article
Modulating the Expression of Disease Genes with RNA-Based Therapy.
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- PLoS Genetics, 2007, v. 3, n. 6, p. e109, doi. 10.1371/journal.pgen.0030109
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- Publication type:
- Article
Targeting the DP2 receptor alleviates muscle atrophy and diet‐induced obesity in mice through oxidative myofiber transition.
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- Journal of Cachexia, Sarcopenia & Muscle, 2023, v. 14, n. 1, p. 342, doi. 10.1002/jcsm.13136
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- Publication type:
- Article
RNA-targeted splice-correction therapy for neuromuscular disease.
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- Brain: A Journal of Neurology, 2010, v. 133, n. 4, p. 957, doi. 10.1093/brain/awq002
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- Publication type:
- Article
Improved cell-penetrating peptide–PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle.
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- Nucleic Acids Research, 2008, v. 36, n. 20, p. 6418, doi. 10.1093/nar/gkn671
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- Publication type:
- Article
Metformin sensitizes sorafenib to inhibit postoperative recurrence and metastasis of hepatocellular carcinoma in orthotopic mouse models.
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- Journal of Hematology & Oncology, 2016, v. 9, p. 1, doi. 10.1186/s13045-016-0253-6
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- Publication type:
- Article
Correction: Wild-Type Mouse Models to Screen Antisense Oligonucleotides for Exon-Skipping Efficacy in Duchenne Muscular Dystrophy.
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- 2018
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- Correction Notice
Correction: Intrahepatic Tissue Implantation Represents a Favorable Approach for Establishing Orthotopic Transplantation Hepatocellular Carcinoma Mouse Models.
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- 2018
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- Publication type:
- Correction Notice
Delivery of siRNA to the mouse brain by systemic injection of targeted exosomes.
- Published in:
- Nature Biotechnology, 2011, v. 29, n. 4, p. 341, doi. 10.1038/nbt.1807
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- Publication type:
- Article
fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice.
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- Human Molecular Genetics, 2019, v. 28, n. 4, p. 699, doi. 10.1093/hmg/ddy377
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- Publication type:
- Article
Generalizable anchor aptamer strategy for loading nucleic acid therapeutics on exosomes.
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- EMBO Molecular Medicine, 2024, v. 16, n. 4, p. 1027, doi. 10.1038/s44321-024-00049-7
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- Publication type:
- Article
MOTS‐c promotes phosphorodiamidate morpholino oligomer uptake and efficacy in dystrophic mice.
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- EMBO Molecular Medicine, 2021, v. 13, n. 2, p. 1, doi. 10.15252/emmm.202012993
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- Publication type:
- Article
Intrahepatic Tissue Implantation Represents a Favorable Approach for Establishing Orthotopic Transplantation Hepatocellular Carcinoma Mouse Models.
- Published in:
- PLoS ONE, 2016, v. 11, n. 1, p. 1, doi. 10.1371/journal.pone.0148263
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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