Ex Vivo COL7A1 Correction for Recessive Dystrophic Epidermolysis Bullosa Using CRISPR/Cas9 and Homology-Directed Repair

• 主要な著者: Araksya Izmiryan (著者), Clarisse Ganier (著者), Matteo Bovolenta (著者), Alain Schmitt (著者), Fulvio Mavilio (著者), Alain Hovnanian (著者)
• フォーマット: 図書
• 出版事項: Elsevier, 2018-09-01T00:00:00Z.
• 主題: article
• オンライン･アクセス: Connect to this object online.

Recessive dystrophic epidermolysis bullosa is a rare and severe genetic skin disease resulting in blistering of the skin and mucosa. Recessive dystrophic epidermolysis bullosa (RDEB) is caused by a wide variety of mutations in COL7A1-encoding type VII collagen, which is essential for dermal-epiderma...