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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Gepubliceerd in:Mol Ther Nucleic Acids
Hoofdauteurs: Bremer, Jeroen, van der Heijden, Elisabeth H., Eichhorn, Daryll S., Meijer, Rowdy, Lemmink, Henny H., Scheffer, Hans, Sinke, Richard J., Jonkman, Marcel F., Pasmooij, Anna M.G., Van den Akker, Peter C.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: American Society of Gene & Cell Therapy 2019
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Article
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6831832/
https://ncbi.nlm.nih.gov/pubmed/31670143
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2019.09.009
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