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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...
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 |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Elsevier
2023
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Materias: | |
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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