Antisense Oligonucleotide-mediated Exon Skipping as a Systemic Therapeutic Approach for Recessive Dystrophic Epidermolysis Bullosa

Antisense Oligonucleotide-mediated Exon Skipping as a Systemic Therapeutic Approach for Recessive Dystrophic Epidermolysis Bullosa

The “generalized severe” form of recessive dystrophic epidermolysis bullosa (RDEB-gen sev) is caused by bi-allelic null mutations in COL7A1, encoding type VII collagen. The absence of type VII collagen leads to blistering of the skin and mucous membranes upon the slightest trauma. Because most patie...

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Bibliographic Details
Main Authors: Jeroen Bremer, Olivier Bornert, Alexander Nyström, Antoni Gostynski, Marcel F Jonkman, Annemieke Aartsma-Rus, Peter C van den Akker, Anna MG Pasmooij
Format: Article
Language:English
Published: Elsevier 2016-01-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
antisense oligonucleotide
COL7A1
exon skipping
recessive dystrophic epidermolysis bullosa
therapy
type VII collagen
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253117301063

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http://www.sciencedirect.com/science/article/pii/S2162253117301063

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