Found: 15
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Muscle regeneration affects Adeno Associated Virus 1 mediated transgene transcription.
- Published in:
- Scientific Reports, 2022, v. 12, n. 1, p. 1, doi. 10.1038/s41598-022-13405-9
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- Article
Allele‐specific silencing therapy for Dynamin 2‐related dominant centronuclear myopathy.
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- EMBO Molecular Medicine, 2018, v. 10, n. 2, p. 239, doi. 10.15252/emmm.201707988
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- Article
Delivery is key: lessons learnt from developing splice-switching antisense therapies.
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- EMBO Molecular Medicine, 2017, v. 9, n. 5, p. 545, doi. 10.15252/emmm.201607199
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- Article
Rescue of cardiomyopathy through U7sn RNA-mediated exon skipping in Mybpc3-targeted knock-in mice.
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- EMBO Molecular Medicine, 2013, v. 5, n. 7, p. 1060, doi. 10.1002/emmm.201202168
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- Article
Dystrophin rescue by trans-splicing: a strategy for DMD genotypes not eligible for exon skipping approaches.
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- Nucleic Acids Research, 2013, v. 41, n. 17, p. 8391, doi. 10.1093/nar/gkt621
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- Article
Repair of Rhodopsin mRNA by Spliceosome-Mediated RNA Trans-Splicing: A New Approach for Autosomal Dominant Retinitis Pigmentosa.
- Published in:
- Molecular Therapy, 2015, v. 23, n. 5, p. 918, doi. 10.1038/mt.2015.11
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- Article
Intrinsic Transgene Immunogenicity Gears CD8<sup>+</sup> T-cell Priming After rAAV-Mediated Muscle Gene Transfer.
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- Molecular Therapy, 2015, v. 23, n. 4, p. 697, doi. 10.1038/mt.2014.235
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- Article
AAV Genome Loss From Dystrophic Mouse Muscles During AAV-U7 snRNA-mediated Exon-skipping Therapy.
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- Molecular Therapy, 2013, v. 21, n. 8, p. 1551, doi. 10.1038/mt.2013.121
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- Article
Muscle Function Recovery in Golden Retriever Muscular Dystrophy After AAV1-U7 Exon Skipping.
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- Molecular Therapy, 2012, v. 20, n. 11, p. 2120, doi. 10.1038/mt.2012.181
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- Article
Correction of the Middle Eastern M712T Mutation Causing GNE Myopathy by Trans-Splicing.
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- NeuroMolecular Medicine, 2014, v. 16, n. 2, p. 322, doi. 10.1007/s12017-013-8278-2
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- Article
Abnormal splicing switch of DMD's penultimate exon compromises muscle fibre maintenance in myotonic dystrophy.
- Published in:
- Nature Communications, 2015, v. 6, n. 5, p. 7205, doi. 10.1038/ncomms8205
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- Article
Antisense pre-treatment increases gene therapy efficacy in dystrophic muscles.
- Published in:
- Human Molecular Genetics, 2016, v. 25, n. 16, p. 3555, doi. 10.1093/hmg/ddw201
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- Article
Dysferlin rescue by spliceosome-mediated pre-mRNA trans-splicing targeting introns harbouring weakly defined 3' splice sites.
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- Human Molecular Genetics, 2015, v. 24, n. 14, p. 4049, doi. 10.1093/hmg/ddv141
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- Article
Transient Immunomodulation Allows Repeated Injections of AAV1 and Correction of Muscular Dystrophy in Multiple Muscles.
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- Molecular Therapy, 2008, v. 16, n. 3, p. 541, doi. 10.1038/sj.mt.6300377
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- Article
HIRA, a mammalian homologue of Saccharomyces cerevisiae transcriptional co-repressors, interacts with Pax3.
- Published in:
- Nature Genetics, 1998, v. 20, n. 1, p. 74, doi. 10.1038/1739
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- Article