Improved Double-Nicking Strategies for COL7A1-Editing by Homologous Recombination

• Hoofdauteurs: Thomas Kocher (Auteur), Roland N. Wagner (Auteur), Alfred Klausegger (Auteur), Christina Guttmann-Gruber (Auteur), Stefan Hainzl (Auteur), Johann W. Bauer (Auteur), Julia Reichelt (Auteur), Ulrich Koller (Auteur)
• Formaat: Boek
• Gepubliceerd in: Elsevier, 2019-12-01T00:00:00Z.
• Onderwerpen: article
• Online toegang: Connect to this object online.

Current gene-editing approaches for treatment of recessive dystrophic epidermolysis bullosa (RDEB), an inherited, severe form of blistering skin disease, suffer from low efficiencies and safety concerns that complicate implementation in clinical settings. We present a strategy for efficient and prec...