Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine...
Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD
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London: Nature Publishing Group UK
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English
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London: Nature Publishing Group UK
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Manipulation of pre-mRNA splicing by antisense oligonucleotides (AOs) offers considerable potential for a number of genetic disorders. One of these is Duchenne muscular dystrophy (DMD), where mutations in the dystrophin gene typically result in premature termination of translation that causes a loss of functional protein. AOs can induce exon skippi...
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Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD
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TN_cdi_proquest_miscellaneous_68860874
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_68860874
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ISSN
0969-7128
E-ISSN
1476-5462
DOI
10.1038/sj.gt.3302800
