Gene editing is suitable to treat GM1 Gangliosidosis: a proof-of-concept study
Gene editing is suitable to treat GM1 Gangliosidosis: a proof-of-concept study
About this item
Full title
Author / Creator
Leclerc, Delphine , Goujon, Louise , Jaillard, Sylvie , Nouyou, Benedicte , Cluzeau, Laurence , Damaj, Lena , Dubourg, Christele , Etcheverry, Amandine , Levade, Thierry , Froissart, Roseline , Dreano, Stephane , Guillory, Xavier , Eriksson, Leif A , Launay, Erika , Mouriaux, Frederic , Marc-Antoine Belaud-Rotureau , Odent, Sylvie and Gilot, David
Publisher
Cold Spring Harbor: Cold Spring Harbor Laboratory Press
Journal title
Language
English
Formats
Publication information
Publisher
Cold Spring Harbor: Cold Spring Harbor Laboratory Press
Subjects
More information
Scope and Contents
Contents
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 beta-galactosidase (β-gal) activity, leading to neurodegeneration associated with premature death. Currently, no effective therapy for GM1 ganglio...
Alternative Titles
Full title
Gene editing is suitable to treat GM1 Gangliosidosis: a proof-of-concept study
Authors, Artists and Contributors
Author / Creator
Goujon, Louise
Jaillard, Sylvie
Nouyou, Benedicte
Cluzeau, Laurence
Damaj, Lena
Dubourg, Christele
Etcheverry, Amandine
Levade, Thierry
Froissart, Roseline
Dreano, Stephane
Guillory, Xavier
Eriksson, Leif A
Launay, Erika
Mouriaux, Frederic
Marc-Antoine Belaud-Rotureau
Odent, Sylvie
Gilot, David
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_proquest_journals_2651431522
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_journals_2651431522
Other Identifiers
E-ISSN
2692-8205
DOI
10.1101/2022.04.17.488473
How to access this item
https://www.proquest.com/docview/2651431522?pq-origsite=primo&accountid=13902
