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...

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Bibliographic Details
Published in:Mol Ther Nucleic Acids
Main Authors: Kocher, Thomas, Wagner, Roland N., Klausegger, Alfred, Guttmann-Gruber, Christina, Hainzl, Stefan, Bauer, Johann W., Reichelt, Julia, Koller, Ulrich
Format: Artigo
Language:Inglês
Published: American Society of Gene & Cell Therapy 2019
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Article
Online Access: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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