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

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...

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Detalles Bibliográficos
Autores principales: Kocher, Thomas, Wagner, Roland N., Klausegger, Alfred, Guttmann-Gruber, Christina, Hainzl, Stefan, Bauer, Johann W., Reichelt, Julia, Koller, Ulrich
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6838546/
https://www.ncbi.nlm.nih.gov/pubmed/31670199
http://dx.doi.org/10.1016/j.omtn.2019.09.011