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. PubMed Article
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. PubMed Article
Plasmids from Article
| ID | Plasmid | Purpose |
|---|---|---|
| 190190 | pWPT-/mEGFP-1T-IRES-mCherry | Lentiviral expression vector with altered Kozak sequence to direct EGFP expression. mCherry expression is regulated by an IRES. |
| 190193 | pUC19-sgNCF1 | Expresses a gRNA for base editing the Kozak sequence of the human NCF1 gene |
| 190605 | pWPT-mCherry | Obtained by cloning 5 stop codons in place of the EGFP Kozak sequence in pWPT-/mEGFP-1T-IRES-mCherry |
| 190606 | pWPT-mEGFP | Obtained by creating a deletion inside the mCherry coding sequence in pWPT-/GCCACC-mEGFP-IRES-mCherry (Addgene #49235) |
| 190607 | pUC19-sg-1 | Expresses a gRNA for base editing the EGFP Kozak sequence of pWPT-/mEGFP-1T-IRES-mCherry |