An RNA-targeted therapy for dystrophic epidermolysis bullosa

Functional impairment or complete loss of type VII collagen, caused by mutations within COL7A1, lead to the severe recessive form of the skin blistering disease dystrophic epidermolysis bullosa (RDEB). Here, we successfully demonstrate RNA trans-splicing as an auspicious repair option for mutations...

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Detalles Bibliográficos
Autores principales: Peking, Patricia, Koller, Ulrich, Duarte, Blanca, Murillas, Rodolfo, Wolf, Susanne, Maetzig, Tobias, Rothe, Michael, Kocher, Thomas, García, Marta, Brachtl, Gabriele, Schambach, Axel, Larcher, Fernando, Reichelt, Julia, Bauer, Johann W., Murauer, Eva M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2017
Materias:
RNA and RNA-protein complexes
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5737646/
https://www.ncbi.nlm.nih.gov/pubmed/28973459
http://dx.doi.org/10.1093/nar/gkx669

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