Engineering synthetic and recombinant human lysosomal β-glucocerebrosidase for enzyme replacement therapy for Gaucher disease

Engineering synthetic and recombinant human lysosomal β-glucocerebrosidase for enzyme replacement therapy for Gaucher disease

Gaucher Disease (GD) is an autosomal recessive, lysosomal storage disease caused by pathogenic variants in the glucocerebrosidase gene, leading to the loss of β-glucocerebrosidase (GCase) enzymatic activity. Enzyme replacement therapy (ERT) with recombinant GCase is the standard of care in GD patien...

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Detalhes bibliográficos
Principais autores: Figueiredo, Lílian L. S., Junior, Wilson L., da Silva Goncalves, Victor W., Ramos, Ester S., D’Almeida, Vania, de Souza, Lucas E. B., Orellana, Maristela D., Abraham, Kuruvilla J., Lichtenstein, Flávio, Bleicher, Lucas
Outros Autores: Massachusetts Institute of Technology. Synthetic Biology Center
Formato: Artigo
Idioma:English
Publicado em: Springer International Publishing 2024
Acesso em linha:https://hdl.handle.net/1721.1/157396

Internet

https://hdl.handle.net/1721.1/157396

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