Personalized Development of Antisense Oligonucleotides for Exon Skipping Restores Type XVII Collagen Expression in Junctional Epidermolysis Bullosa

Intermediate junctional epidermolysis bullosa caused by mutations in the COL17A1 gene is characterized by the frequent development of blisters and erosions on the skin and mucous membranes. The rarity of the disease and the heterogeneity of the underlying mutations renders therapy developments chall...

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Publicado en:Int J Mol Sci
Main Authors: Ablinger, Michael, Lettner, Thomas, Friedl, Nicole, Potocki, Hannah, Palmetzhofer, Theresa, Koller, Ulrich, Illmer, Julia, Liemberger, Bernadette, Hainzl, Stefan, Klausegger, Alfred, Reisenberger, Manuela, Lambert, Jo, Van Gele, Mireille, Desmet, Eline, Van Maelsaeke, Els, Wimmer, Monika, Zauner, Roland, Bauer, Johann W., Wally, Verena
Formato: Artigo
Idioma:Inglês
Publicado: MDPI 2021
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Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC8036626/
https://ncbi.nlm.nih.gov/pubmed/33805154
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms22073326
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