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Plasmid containing a CMV-driven NLS-dAcCas12n(D240)-ABE-Design3-NLS cassette and a U6-driven sgRNA_V6 cassette(containing BsaI sites for spacer insertion).
Plasmid containing a CMV-driven NLS-AcCas12n-NLS (human-codonoptimized) cassette and a U6-driven sgRNA_V6 cassette (containing BsaI sites for spacer insertion). Carbenicillin resistance.
Expresses dSa Cas9 fused to gold standard VP64 activator domain and Sa sgRNA for Hbb promoter, vector allows for activation of mouse Hbb, can be packaged and delivered as AAV