Gene editing is suitable to treat GM1 Gangliosidosis: a proof-of-concept study
Delphine Leclerc, Louise Goujon, Sylvie Jaillard, Bénédicte Nouyou, Laurence Cluzeau, Léna Damaj, Christèle Dubourg, Amandine Etcheverry, Thierry Levade, Roseline Froissart, Stéphane Dréano, X. Guillory, Leif Axel Eriksson, Erika Launay, F. Mouriaux, , David Gilot
This study shows that adenine base editors can correct 41% of GLB1 pathogenic SNVs in patient-derived fibroblasts, restoring β-gal activity without significant off-target effects.
bioRxiv · Apr 17, 2022 · PreprintGenetics