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...

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Bibliographische Detailangaben
Hauptverfasser:	ILDAR, Gainetdinov, IBRAHEIM, Raed, SONTHEIMER, Erik Joseph, EDRAKI, Alireza, XUE, Wen, MIR, Aamir
Format:	Patent
Sprache:	eng
Schlagworte:	BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA ENZYMOLOGY METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPIRITS VINEGAR WINE
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Beschreibung
Zusammenfassung:	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 RNA. sequences that permit a complete sgRNA-Nme1Cas9 vector to be inserted into an adeno-associated viral plasmid that is compatible for in vivo administration. Furthermore, Type II-C Cas9 oithologs have been identified that target protospacer adjacent motif sequences limited to between one - four required nucleotides.