Targeted CRISPR delivery platforms

The present invention is related to compositions and methods for gene therapy. Several approaches described herein utilize the Neisseria meningitidis Cas9 system that provides a hvperaccurate CRISPR gene editing platform. Furthermore, the invention incorporates full length and truncated single guide...

Ausführliche Beschreibung

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Bibliographische Detailangaben
Hauptverfasser: ILDAR, Gainetdinov, IBRAHEIM, Raed, SONTHEIMER, Erik Joseph, EDRAKI, Alireza, XUE, Wen, MIR, Aamir
Format: Patent
Sprache:eng
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