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FLT201: An AAV-mediated gene therapy for type 1 Gaucher disease designed to target difficult to reach tissues

Romuald Corbau,Carlos Miranda,Fabrizio Comper,Petya Kalcheva,Elisa Chisari,Clement Cocita,Samantha Correia,Jalpa Pandya,Benjamin Liou,Natalie Northcott,Emmaline Stotter,Erald Shehu,I-Mei Yu,Jaminder Khinder,Rose Sheridan,Ying Sun,Amit Nathwani

Molecular Genetics and Metabolism（2021）

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Gaucher Disease
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