Article

Engineering Synthetic and Recombinant Human Lysosomal b-Glucocerebrosidase for Enzyme Replacement Therapy for Gaucher Disease

2024-07-19

Abstract excerpt

<title>Abstract</title> <p>Gaucher Disease (GD) is an autosomal recessive, lysosomal storage disease caused by pathogenic variants in the glucocerebrosidase gene, leading to the loss of b-glucocerebrosidase (GCase) enzymatic activity. Enzyme replacement therapy (ERT) with recombinant GCase is the standard of care in GD patients. Our study investigates the combined use of <italic>in silico</italic> molecular evolu...

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Literature Corpus work: 2bb63f66-1ddd-5d61-9c55-16bafcb39e1a
DOI: 10.21203/rs.3.rs-4625596/v1

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