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Dystrophin rescue by trans-splicing: a strategy for DMD genotypes not eligible for exon skipping approaches.
- Published in:
- Nucleic Acids Research, 2013, v. 41, n. 17, p. 8391, doi. 10.1093/nar/gkt621
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- Article
Exon Exchange Approach to Repair Duchenne Dystrophin Transcripts.
- Published in:
- PLoS ONE, 2010, v. 5, n. 5, p. 1, doi. 10.1371/journal.pone.0010894
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- Publication type:
- Article
AAV Genome Loss From Dystrophic Mouse Muscles During AAV-U7 snRNA-mediated Exon-skipping Therapy.
- Published in:
- 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.
- Published in:
- Molecular Therapy, 2012, v. 20, n. 11, p. 2120, doi. 10.1038/mt.2012.181
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- Publication type:
- 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