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Human genetic variation alters CRISPR-Cas9 on- and off-targeting specificity at therapeutically implicated loci

The CRISPR-Cas9 nuclease system holds enormous potential for therapeutic genome editing of a wide spectrum of diseases. Large efforts have been made to further understanding of on- and off-target activity to assist the design of CRISPR-based therapies with optimized efficacy and safety. However, cur...

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Bibliographische Detailangaben
Veröffentlicht in:Proc Natl Acad Sci U S A
Hauptverfasser: Lessard, Samuel, Francioli, Laurent, Alfoldi, Jessica, Tardif, Jean-Claude, Ellinor, Patrick T., MacArthur, Daniel G., Lettre, Guillaume, Orkin, Stuart H., Canver, Matthew C.
Format: Artigo
Sprache:Inglês
Veröffentlicht: National Academy of Sciences 2017
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Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5748207/
https://ncbi.nlm.nih.gov/pubmed/29229813
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1714640114
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