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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Foilsithe in: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.
Formáid: Artigo
Teanga:Inglês
Foilsithe: Oxford University Press 2017
Ábhair:
RNA and RNA-protein complexes
Rochtain Ar Líne: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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