A NOVEL SYNTHETIC AND RECOMBINANT GLUCOCEREBROSIDASE FOR GAUCHER DISEASE: IN SILICO MOLECULAR EVOLUTION AND GENE THERAPY APPROACHES TO ENHANCE ENZYME ACTIVITY

A NOVEL SYNTHETIC AND RECOMBINANT GLUCOCEREBROSIDASE FOR GAUCHER DISEASE: IN SILICO MOLECULAR EVOLUTION AND GENE THERAPY APPROACHES TO ENHANCE ENZYME ACTIVITY

Background: Gaucher disease (GD) is a rare lysosomal storage disease caused by pathogenic variants in the glucocerebrosidase gene (GBA1) resulting in a markedly decreased activity of the lysosomal enzyme b-glucocerebrosidase (GCase). Enzyme replacement therapy is the gold standard for treating GD pa...

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Bibliographic Details
Main Authors: LLS Figueiredo, LEB Souza, MD Orellana, DT Covas, F Lichtenstein, V Siciliano, KJ Abraham, R Weiss, SL Gerson, AM Fontes
Format: Article
Language:English
Published: Elsevier 2023-10-01
Series:Hematology, Transfusion and Cell Therapy
Online Access:http://www.sciencedirect.com/science/article/pii/S2531137923016887

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http://www.sciencedirect.com/science/article/pii/S2531137923016887

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