FLT201, a novel liver-directed AAV gene therapy candidate for Gaucher disease type 1.
FLT201 是一种新型的肝脏靶向 AAV 基因治疗候选药物，用于治疗 1 型戈谢病。
期刊:Molecular Therapy
影响因子:12
doi:10.1016/j.ymthe.2025.05.003
Comper Fabrizio, Miranda Carlos J, Liou Benjamin, Dodev Tihomir, Jeyakumar Jey M, Canavese Miriam, Cocita Clement, Khoshrou Khashayar, Tiscornia Gustavo, Chisari Elisa, Stotter Emmaline, Shehu Erald, Sridharan Sudharsan, Yu I-Mei, Pandya Jalpa, Khinder Jaminder, Northcott Natalie, Kalcheva Petya, Correia Samantha, Sun Ying, Dane Allison P, Sheridan Rose, Nathwani Amit C, Corbau Romuald
