A non-viral and selection-free COL7A1 HDR approach with improved safety profile for dystrophic epidermolysis bullosa

• Main Authors: Thomas Kocher (Author), Johannes Bischof (Author), Simone Alexandra Haas (Author), Oliver Patrick March (Author), Bernadette Liemberger (Author), Stefan Hainzl (Author), Julia Illmer (Author), Anna Hoog (Author), Katharina Muigg (Author), Heide-Marie Binder (Author), Alfred Klausegger (Author), Dirk Strunk (Author), Johann Wolfgang Bauer (Author), Toni Cathomen (Author), Ulrich Koller (Author)
• Format: Book
• Published: Elsevier, 2021-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Gene editing via homology-directed repair (HDR) currently comprises the best strategy to obtain perfect corrections for pathogenic mutations of monogenic diseases, such as the severe recessive dystrophic form of the blistering skin disease epidermolysis bullosa (RDEB). Limitations of this strategy,...