Galactosialidosis: preclinical enzyme replacement therapy in a mouse model of the disease, a proof of concept

Galactosialidosis is a rare lysosomal storage disease caused by a congenital defect of protective protein/cathepsin A (PPCA) and secondary deficiency of neuraminidase-1 and β-galactosidase. PPCA is a lysosomal serine carboxypeptidase that functions as a chaperone for neuraminidase-1 and β-galactosid...

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Detalhes bibliográficos
Publicado no:Mol Ther Methods Clin Dev
Main Authors: Cadaoas, Jaclyn, Hu, Huimin, Boyle, Gabrielle, Gomero, Elida, Mosca, Rosario, Jayashankar, Kartika, Machado, Mike, Cullen, Sean, Guzman, Belle, van de Vlekkert, Diantha, Annunziata, Ida, Vellard, Michel, Kakkis, Emil, Koppaka, Vish, d’Azzo, Alessandra
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2020
Assuntos:
Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7782203/
https://ncbi.nlm.nih.gov/pubmed/33426146
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.11.012
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