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Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers.
- Published in:
- Nature Medicine, 2015, v. 21, n. 3, p. 270, doi. 10.1038/nm.3765
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- Article
Regeneration of a well-differentiated human airway surface epithelium by spheroid and lentivirus vector-transduced airway cells.
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- Journal of Gene Medicine, 2004, v. 6, n. 8, p. 846, doi. 10.1002/jgm.570
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- Article
In Vitro Correction of a Pseudoexon-Generating Deep Intronic Mutation in LGMD2A by Antisense Oligonucleotides and Modified Small Nuclear RNAs.
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- Human Mutation, 2013, v. 34, n. 10, p. 1387, doi. 10.1002/humu.22379
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- Article
Efficient bypass of mutations in dysferlin deficient patient cells by antisense-induced exon skipping.
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- Human Mutation, 2010, v. 31, n. 2, p. 136, doi. 10.1002/humu.21160
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- Publication type:
- Article
A focus on dominant negative variants in a series of 170 heterozygous FXI‐deficient patients.
- Published in:
- Haemophilia, 2023, v. 29, n. 4, p. 1113, doi. 10.1111/hae.14802
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- Article