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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Detalles Bibliográficos
Autores principales: Kocher, Thomas, Bischof, Johannes, Haas, Simone Alexandra, March, Oliver Patrick, Liemberger, Bernadette, Hainzl, Stefan, Illmer, Julia, Hoog, Anna, Muigg, Katharina, Binder, Heide-Marie, Klausegger, Alfred, Strunk, Dirk, Bauer, Johann Wolfgang, Cathomen, Toni, Koller, Ulrich
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8368800/
https://www.ncbi.nlm.nih.gov/pubmed/34458008
http://dx.doi.org/10.1016/j.omtn.2021.05.015

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