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.
Ambrosini C, et al. Among authors: kheir e.
Nucleic Acids Res. 2022 Oct 14;50(18):10756-10771. doi: 10.1093/nar/gkac799.
Nucleic Acids Res. 2022.
PMID: 36165847
Free PMC article.