Optimization of systemic AAV9 gene therapy in Niemann-Pick disease type C1 mice
尼曼匹克病 C1 型小鼠全身 AAV9 基因治疗的优化
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doi:10.1101/2024.06.07.597901
Avani V Mylvara, Alana L Gibson, Tansy Gu, Cristin D Davidson, Art A Incao, Katerina Melnyk, Dominick Pierre-Jacques, Stephanie M Cologna, Charles P Venditti, Forbes D Porter, William J Pavan
