CPC A61K 48/0066 (2013.01) [A61K 9/127 (2013.01); A61K 31/7105 (2013.01); A61K 38/465 (2013.01); C07K 14/515 (2013.01); C12N 9/22 (2013.01); C12N 15/11 (2013.01); C12N 15/113 (2013.01); C12N 15/907 (2013.01); C07K 2319/09 (2013.01); C07K 2319/80 (2013.01); C12N 2310/20 (2017.05); C12N 2310/315 (2013.01); C12N 2310/321 (2013.01); C12N 2310/331 (2013.01); C12N 2310/335 (2013.01); C12N 2310/344 (2013.01); C12N 2310/3521 (2013.01); C12N 2800/80 (2013.01)] | 24 Claims |
1. A composition for editing a gene target comprising:
(i) an mRNA encoding a base editor protein comprising a DNA binding domain and a deaminase, wherein the mRNA comprises a sequence having at least 95% sequence identity to SEQ ID NO: 2192, and
(ii) a guide RNA comprising a tracr sequence that serves as a binding scaffold for the base editor protein, and a spacer sequence that corresponds to a protospacer on a target gene;
wherein the target gene is PCSK9.
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