Development of AAV-GBA1 Gene Replacement Therapy via Single-IV-Delivery with a Blood Brain Barrier Penetrant AAV Capsid
Charlotte Chung, Cassie Retzlaff,Jeff Thompson, Alexis Bloedel,Amy Bruce,Abigail Ecker,Joseph Clement, Elisabeth R. Knoll,Brian C. Ezell, Brett Hoffman, Tatiana Knox, Mathieu E. Nonnenmacher, Tyler Moyer, Jing Lin,Kyle Grant,Maneesha Paranjpe,Nilesh Pande,Amy Johnson,Johnny Yao, Chanchal Sadhu, Todd A. Carter MOLECULAR THERAPY（2023）
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