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Autor:
Simone A. Haas, Heide-Marie Binder, Thomas Kocher, Johannes Bischof, Toni Cathomen, Oliver Patrick March, Dirk Strunk, Alfred Klausegger, Johann W. Bauer, Bernadette Liemberger, Anna Hoog, Julia Illmer, Katharina Muigg, Stefan Hainzl, Ulrich Koller
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 25, Iss, Pp 237-250 (2021)
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 epidermoly