Article
QR-313, an Antisense Oligonucleotide, Shows Therapeutic Efficacy for Treatment of Dominant and Recessive Dystrophic Epidermolysis Bullosa: A Preclinical Study.
The Journal of investigative dermatology - 1 Apr 2021
Bornert Olivier, Hogervorst Marieke, Nauroy Pauline, Bischof Johannes, Swildens Jim, Athanasiou Ioannis, Tufa Sara F, Keene Douglas R, Kiritsi Dimitra, Hainzl Stefan, Murauer Eva M, Marinkovich M Peter, Platenburg Gerard, Hausser Ingrid, Wally Verena, Ritsema Tita, Koller Ulrich, Haisma Elisabeth M, Nyström Alexander
Abstract excerpt
Dystrophic epidermolysis bullosa (DEB) is a blistering skin disease caused by mutations in the gene COL7A1 encoding collagen VII. DEB can be inherited as recessive DEB (RDEB) or dominant DEB (DDEB) and is associated with a high wound burden. Perpetual cycles of wounding and healing drive fibrosis in DDEB and RDEB, as well as the formation of a tumor-permissive microenvironment. Prolonging wound-free episodes by...
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