Bernadette Liemberger

TL;DR: This work demonstrates that COL7A1 editing based on precise end joining-mediated DNA repair is an efficient strategy to revert the disease-associated nature of DEB, regardless of the mutational inheritance.

TL;DR: A modified, easily applicable, fluorescence-based screening system for the generation and analysis of antisense molecules specifically capable of improving the RNA reprogramming efficiency of a selected KRT14-specific RNA trans-splicing molecule is described.

TL;DR: Rabbit polyclonal antibodies that recognize either human or murine collagen VII are generated by immunization with homologous peptides and combined sequence alignment and peptide competition binding assays enabled identification of the major antisera recognizing epitopes.

TL;DR: Detailed on- and off-target analyses confirmed that the combined treatment of paired nicking and single-stranded oligonucleotides constituted a highly efficient COL7A1 editing strategy, associated with a significantly improved safety profile, and represent a further advancement in the field of traceless genome editing for genodermatoses.

TL;DR: In this paper, the authors designed and tested three AONs in combination with a cationic liposomal carrier for exon skipping of COL17A1 exon 7 in 2D culture and in 3D skin equivalents.