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5′RNA Trans-Splicing Repair of COL7A1 Mutant Transcripts in Epidermolysis Bullosa

Mutations within the COL7A1 gene underlie the inherited recessive subtype of the blistering skin disease dystrophic epidermolysis bullosa (RDEB). Although gene replacement approaches for genodermatoses are clinically advanced, their implementation for RDEB is challenging and requires endogenous regu...

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Detalles Bibliográficos
Autores principales: Mayr, Elisabeth, Ablinger, Michael, Lettner, Thomas, Murauer, Eva M., Guttmann-Gruber, Christina, Piñón Hofbauer, Josefina, Hainzl, Stefan, Kaiser, Manfred, Klausegger, Alfred, Bauer, Johann W., Koller, Ulrich, Wally, Verena
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8835740/
https://www.ncbi.nlm.nih.gov/pubmed/35163654
http://dx.doi.org/10.3390/ijms23031732