Preclinical Enzyme Replacement Therapy with a Recombinant β-Galactosidase-Lectin Fusion for CNS Delivery and Treatment of GM1-Gangliosidosis

GM1-gangliosidosis is a catastrophic, neurodegenerative lysosomal storage disease caused by a deficiency of lysosomal β-galactosidase (β-Gal). The primary substrate of the enzyme is GM1-ganglioside (GM1), a sialylated glycosphingolipid abundant in nervous tissue. Patients with GM1-gangliosidosis pre...

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Detalles Bibliográficos
Autores principales: Weesner, Jason Andrew, Annunziata, Ida, Yang, Tianhong, Acosta, Walter, Gomero, Elida, Hu, Huimin, van de Vlekkert, Diantha, Ayala, Jorge, Qiu, Xiaohui, Fremuth, Leigh Ellen, Radin, David N., Cramer, Carole L., d’Azzo, Alessandra
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9406850/
https://www.ncbi.nlm.nih.gov/pubmed/36010656
http://dx.doi.org/10.3390/cells11162579

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9406850/
https://www.ncbi.nlm.nih.gov/pubmed/36010656
http://dx.doi.org/10.3390/cells11162579

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