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

• Главные авторы: Gaetano Naso (Автор), Soragia Athina Gkazi (Автор), Christos Georgiadis (Автор), Vignesh Jayarajan (Автор), Joanna Jacków (Автор), Roland Fleck (Автор), Leanne Allison (Автор), Olumide Kayode Ogunbiyi (Автор), John Alexander McGrath (Автор), Dusko Ilic (Автор), Wei-Li Di (Автор), Anastasia Petrova (Автор), Waseem Qasim (Автор)
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• Опубликовано: Elsevier, 2023-05-01T00:00:00Z.
• Предметы: article
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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...