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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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Gorde:
Xehetasun bibliografikoak
Argitaratua izan da:Int J Mol Sci
Egile Nagusiak: 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
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: MDPI 2021
Gaiak:
Sarrera elektronikoa: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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