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Development of a Lentiviral Vector for High-Yield Production of Synthetic and Recombinant GCase for Gaucher Disease Therapy

2025-06-17

Abstract excerpt

Gaucher disease (GD) is an autosomal recessive disorder caused by the deficient activity of the lysosomal enzyme glucocerebrosidase (GCase). Although enzyme replacement therapy (ERT) remains the most effective treatment for GD patients, its high cost significantly limits accessibility. To enhance production efficiency, we developed a lentiviral system encoding a codon-optimized GCase gene driven by the human elong...

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Literature Corpus work
fc5bbe60-10ef-5aaa-beb1-e51a4c9d16c4
DOI
10.20944/preprints202506.1350.v1
Open publication

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Development of a Lentiviral Vector for High-Yield Production of Synthetic and Recombinant GCase for Gaucher Disease TherapyDOI 10.20944/preprints202506.1350.v1
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