Gene editing is suitable to treat GM1 Gangliosidosis: a proof-of-concept study

Leclerc, Delphine; Goujon, Louise; Jaillard, Sylvie; Nouyou, Bénédicte; Cluzeau, Laurence; Damaj, Léna; Dubourg, Christèle; Etcheverry, Amandine; Levade, Thierry; Froissart, Roseline; Dréano, Stéphane; Guillory, X.; A. Eriksson, Leif; Launay, Erika; Mouriaux, F.; Belaud‐Rotureau, Marc‐Antoine; Odent, Sylvie; Gilot, David

doi:10.1101/2022.04.17.488473

Public

Gene editing is suitable to treat GM1 Gangliosidosis: a proof-of-concept study

Published in bioRxiv (Cold Spring Harbor Laboratory) • Apr 17, 2022

Authors:

Delphine Leclerc

Louise Goujon

Sylvie Jaillard

Abstract

Abstract Ganglioside-monosialic acid (GM1) gangliosidosis, a rare autosomal recessive disorder, is frequently caused by deleterious single nucleotide variants (SNVs) in GLB1 gene. These variants result in reduced β-galactosidase (β-gal) activity, leading to neurodegeneration associated with prematur...

Subject

Genome editing

Genetic enhancement

Gene

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