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Translational enhancement by base editing of the Kozak sequence rescues haploinsufficiency

A variety of single-gene human diseases are caused by haploinsufficiency, a genetic condition by which mutational inactivation of one allele leads to reduced protein levels and functional impairment. Translational enhancement of the spare allele could exert a therapeutic effect. Here we developed BO...

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Detalles Bibliográficos
Autores principales:	Ambrosini, Chiara, Destefanis, Eliana, Kheir, Eyemen, Broso, Francesca, Alessandrini, Federica, Longhi, Sara, Battisti, Nicolò, Pesce, Isabella, Dassi, Erik, Petris, Gianluca, Cereseto, Anna, Quattrone, Alessandro
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Oxford University Press 2022
Materias:	Synthetic Biology and Bioengineering
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9561285/ https://www.ncbi.nlm.nih.gov/pubmed/36165847 http://dx.doi.org/10.1093/nar/gkac799

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