FLT201, a novel liver-directed AAV gene therapy candidate for Gaucher disease type 1

Fabrizio Comper, Carlos J. Miranda, Benjamin Liou, Tihomir Dodev, Jey M Jeyakumar, Miriam Canavese, Clément Cocita, Khashayar Khoshrou, Gustavo Tiscórnia, Elisa Chisari, Emmaline Stotter, Erald Shehu, Sudharsan Sridharan, I-Mei Yu, Jalpa Pandya, Jaminder Khinder, Natalie Northcott, Petya Kalcheva, Samantha Correia, Ying Sun, Allison Dane, Rose Sheridan, Amit C. Nathwani, Romuald Corbau