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

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

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,...

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Bibliographic Details
Main Authors: Thomas Kocher, Johannes Bischof, Simone Alexandra Haas, Oliver Patrick March, Bernadette Liemberger, Stefan Hainzl, Julia Illmer, Anna Hoog, Katharina Muigg, Heide-Marie Binder, Alfred Klausegger, Dirk Strunk, Johann Wolfgang Bauer, Toni Cathomen, Ulrich Koller
Format: Article
Language:English
Published: Elsevier 2021-09-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
epidermolysis bullosa
primary keratinocytes
primary fibroblasts
CRISPR/Cas9
Cas9 nickase
double-nicking
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253121001311

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http://www.sciencedirect.com/science/article/pii/S2162253121001311

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