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Translational enhancement by base editing of the Kozak sequence rescues haploinsufficiency.

Ambrosini C, Destefanis E, Kheir E, Broso F, Alessandrini F, Longhi S, Battisti N, Pesce I, Dassi E, Petris G, Cereseto A, Quattrone A
Nucleic Acids Res. 2022 Oct 14;50(18):10756-10771. doi: 10.1093/nar/gkac799. (Link opens in a new window) PubMed (Link opens in a new window) Article

Plasmids from Article

ID Plasmid Purpose
190190pWPT-/mEGFP-1T-IRES-mCherryLentiviral expression vector with altered Kozak sequence to direct EGFP expression. mCherry expression is regulated by an IRES.
190193pUC19-sgNCF1Expresses a gRNA for base editing the Kozak sequence of the human NCF1 gene
190605pWPT-mCherryObtained by cloning 5 stop codons in place of the EGFP Kozak sequence in pWPT-/mEGFP-1T-IRES-mCherry
190606pWPT-mEGFPObtained by creating a deletion inside the mCherry coding sequence in pWPT-/GCCACC-mEGFP-IRES-mCherry (Addgene #49235)
190607pUC19-sg-1Expresses a gRNA for base editing the EGFP Kozak sequence of pWPT-/mEGFP-1T-IRES-mCherry

Antibodies from Article