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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Bibliographic Details
Main Authors: 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
Other Authors: Massachusetts Institute of Technology. Synthetic Biology Center
Format: Article
Language:English
Published: Springer International Publishing 2024
Online Access:https://hdl.handle.net/1721.1/157396

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

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