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
Autor: | Amit C. Nathwani, Romuald Corbau, Petya Kalcheva, Emmaline Stotter, Erald Shehu, Jalpa Pandya, Fabrizio Comper, Clement Cocita, I-Mei Yu, Carlos Henrique Miranda, Natalie Northcott, Elisa Chisari, Rose Sheridan, Samantha Correia |
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Rok vydání: | 2021 |
Předmět: | |
Zdroj: | Molecular Genetics and Metabolism. 132:S27-S28 |
ISSN: | 1096-7192 |
Databáze: | OpenAIRE |
Externí odkaz: |