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

CONCLUSIONS: In summary, 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 a stabilization of the epidermis in a xenograft mouse model. This article is protected by copyright. All rights reserved.
PMID: 30099737 [PubMed - as supplied by publisher]

Source: The British Journal of Dermatology - August 12, 2018 Category: Dermatology Authors: Peking P, Breitenbach JS, Ablinger M, Muss WH, Poetschke FJ, Kocher T, Koller U, Hainzl S, Kitzmueller S, Bauer JW, Reichelt J, Lettner T, Wally V Tags: Br J Dermatol Source Type: research