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Combination Antisense Treatment for Destructive Exon Skipping of Myostatin and Open Reading Frame Rescue of Dystrophin in Neonatal mdx Mice.
- Published in:
- Molecular Therapy, 2015, v. 23, n. 8, p. 1341, doi. 10.1038/mt.2015.88
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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
Nuclear poly(A)-binding protein aggregates misplace a pre-mRNA outside of SC35 speckle causing its abnormal splicing.
- Published in:
- Nucleic Acids Research, 2016, v. 44, n. 22, p. 10929, doi. 10.1093/nar/gkw703
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- Article
piggyBac transposons expressing full-length human dystrophin enable genetic correction of dystrophic mesoangioblasts.
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- Nucleic Acids Research, 2016, v. 44, n. 2, p. 744, doi. 10.1093/nar/gkv1464
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- Article
Pharmacological modulation of the ER stress response ameliorates oculopharyngeal muscular dystrophy.
- Published in:
- Human Molecular Genetics, 2019, v. 28, n. 10, p. 1694, doi. 10.1093/hmg/ddz007
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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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- Publication type:
- Article