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a guide molecule capable of complexing with the Cas protein and directing binding of the guide-Cas protein complex to a target polynucleotide

CES1-triggered liver-specific cargo release of CRISPR/Cas9 elements by cationic triadic copolymeric nanoparticles targeting gene editing of PCSK9 for hyperlipidemia amelioration

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In some embodiments, the method further comprises repairing said cleaved or nicked target polynucleotide by homologous recombination with an recombination template polynucleotide, wherein said repair results in a mutation comprising an insertion, deletion, or substitution of one or more nucleotides of said target polynucleotide

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