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Improved Double-Nicking Strategies for COL7A1-Editing by Homologous Recombination

Current gene-editing approaches for treatment of recessive dystrophic epidermolysis bullosa (RDEB), an inherited, severe form of blistering skin disease, suffer from low efficiencies and safety concerns that complicate implementation in clinical settings. We present a strategy for efficient and prec...

詳細記述

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書誌詳細
出版年:Mol Ther Nucleic Acids
主要な著者: Kocher, Thomas, Wagner, Roland N., Klausegger, Alfred, Guttmann-Gruber, Christina, Hainzl, Stefan, Bauer, Johann W., Reichelt, Julia, Koller, Ulrich
フォーマット: Artigo
言語:Inglês
出版事項: American Society of Gene & Cell Therapy 2019
主題:
オンライン・アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC6838546/
https://ncbi.nlm.nih.gov/pubmed/31670199
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2019.09.011
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