An ex vivo RNA trans-splicing strategy to correct human generalized severe epidermolysis bullosa simplex.

P PekingJ S BreitenbachM AblingerW H MussF J PoetschkeT KocherU KollerS HainzlS KitzmuellerJ W BauerJulia ReicheltT LettnerVerena Wally

Published in: The British journal of dermatology (2018)

Our data demonstrate that trans-splicing-mediated mRNA therapy is an effective method for the correction of dominantly inherited KRT14 mutations at the transcriptional level. This results in the rescue of the EBS-gen sev phenotype and stabilization of the epidermis in a xenograft mouse model.

Keyphrases

mouse model
endothelial cells
induced pluripotent stem cells
electronic health record
gene expression
early onset
big data
pluripotent stem cells
binding protein
oxidative stress
bone marrow
cell therapy