Paired nicking-mediated COL17A1 reframing for junctional epidermolysis bullosa

Junctional epidermolysis bullosa (JEB) is a debilitating hereditary skin disorder caused by mutations in genes encoding laminin-332, type XVII collagen (C17), and integrin-α6β4, which maintain stability between the dermis and epidermis. We designed patient-specific Cas9-nuclease- and -nickase-based...

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Detalles Bibliográficos
Autores principales: Bischof, Johannes, March, Oliver Patrick, Liemberger, Bernadette, Haas, Simone Alexandra, Hainzl, Stefan, Petković, Igor, Leb-Reichl, Victoria, Illmer, Julia, Korotchenko, Evgeniia, Klausegger, Alfred, Hoog, Anna, Binder, Heide-Marie, Garcia, Marta, Duarte, Blanca, Strunk, Dirk, Larcher, Fernando, Reichelt, Julia, Guttmann-Gruber, Christina, Wally, Verena, Hofbauer, Josefina Piñón, Bauer, Johann Wolfgang, Cathomen, Toni, Kocher, Thomas, Koller, Ulrich
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9372311/
https://www.ncbi.nlm.nih.gov/pubmed/35490295
http://dx.doi.org/10.1016/j.ymthe.2022.04.020

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