Cytosine Deaminase Base Editing to Restore COL7A1 in Dystrophic Epidermolysis Bullosa Human: Murine Skin Model

Recessive dystrophic epidermolysis bullosa is a debilitating blistering skin disorder caused by loss-of-function mutations in COL7A1, which encodes type VII collagen, the main component of anchoring fibrils at the dermal−epidermal junction. Although conventional gene therapy approaches through viral...

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Detalles Bibliográficos
Autores principales: Naso, Gaetano, Gkazi, Soragia Athina, Georgiadis, Christos, Jayarajan, Vignesh, Jacków, Joanna, Fleck, Roland, Allison, Leanne, Ogunbiyi, Olumide Kayode, McGrath, John Alexander, Ilic, Dusko, Di, Wei-Li, Petrova, Anastasia, Qasim, Waseem
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10193018/
https://www.ncbi.nlm.nih.gov/pubmed/37213713
http://dx.doi.org/10.1016/j.xjidi.2023.100191

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