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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Detalhes bibliográficos
Publicado no:Nucleic Acids Res
Main Authors: 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: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 2017
Assuntos:
RNA and RNA-protein complexes
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5737646/
https://ncbi.nlm.nih.gov/pubmed/28973459
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gkx669
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