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Generation of β-Glucocerebrosidase variants with increased half-life in human plasma for liver directed AAV gene therapy aimed at the treatment of Gaucher disease type 1

Fabrizio Comper,I-Mei Yu,Petya Kalcheva,Carlos J. Miranda,Elisa Chisari,Clement Cocita,Samantha Correia,Jalpa Pandya,Natalie Northcott,Emmaline Stotter,Erald Shehu,Rose Sheridan,Amit C. Nathwani,Romuald Corbau

Molecular Genetics and Metabolism（2021）

Cited 1|Views25
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Key words
Glucocerebrosidase Mutations,Gaucher Disease
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