Natural Exon Skipping Sets the Stage for Exon Skipping as Therapy for Dystrophic Epidermolysis Bullo...
Natural Exon Skipping Sets the Stage for Exon Skipping as Therapy for Dystrophic Epidermolysis Bullosa
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Publisher
United States: Elsevier Inc
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Language
English
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Publisher
United States: Elsevier Inc
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Dystrophic epidermolysis bullosa (DEB) is a devastating blistering disease affecting skin and mucous membranes. It is caused by pathogenic variants in the COL7A1 gene encoding type VII collagen, and can be inherited dominantly or recessively. Recently, promising proof-of-principle has been shown for antisense oligonucleotide (AON)-mediated exon ski...
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Full title
Natural Exon Skipping Sets the Stage for Exon Skipping as Therapy for Dystrophic Epidermolysis Bullosa
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TN_cdi_doaj_primary_oai_doaj_org_article_a5afe6acdf434eada2678f9c57426506
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_a5afe6acdf434eada2678f9c57426506
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ISSN
2162-2531
E-ISSN
2162-2531
DOI
10.1016/j.omtn.2019.09.009
