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

• Main Authors: Gaetano Naso (Author), Soragia Athina Gkazi (Author), Christos Georgiadis (Author), Vignesh Jayarajan (Author), Joanna Jacków (Author), Roland Fleck (Author), Leanne Allison (Author), Olumide Kayode Ogunbiyi (Author), John Alexander McGrath (Author), Dusko Ilic (Author), Wei-Li Di (Author), Anastasia Petrova (Author), Waseem Qasim (Author)
• 格式: 圖書
• 出版: Elsevier, 2023-05-01T00:00:00Z.
• 主題: article
• 在線閱讀: Connect to this object online.

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...