GeneticsbioRxivPreprint
Gene editing is suitable to treat GM1 Gangliosidosis: a proof-of-concept study
Delphine Leclerc, Louise Goujon, Sylvie Jaillard…·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.
2 citations