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Natural Exon Skipping Sets the Stage for Exon Skipping as Therapy for Dystrophic Epidermolysis Bullosa

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...

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Main Authors: Jeroen Bremer (Author), Elisabeth H. van der Heijden (Author), Daryll S. Eichhorn (Author), Rowdy Meijer (Author), Henny H. Lemmink (Author), Hans Scheffer (Author), Richard J. Sinke (Author), Marcel F. Jonkman (Author), Anna M.G. Pasmooij (Author), Peter C. Van den Akker (Author)
Format: Book
Published: Elsevier, 2019-12-01T00:00:00Z.
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