Waning efficacy in a long-term AAV-mediated gene therapy study in the murine model of Krabbe disease
针对克拉伯病小鼠模型的长期 AAV 介导基因治疗研究的疗效逐渐减弱
期刊:Molecular Therapy
影响因子:12.1
doi:10.1016/j.ymthe.2021.01.026
Gregory J Heller, Michael S Marshall, Yazan Issa, Jeffrey N Marshall, Duc Nguyen, Emily Rue, Koralege C Pathmasiri, Miriam S Domowicz, Richard B van Breemen, Leon M Tai, Stephanie M Cologna, Stephen J Crocker, Maria I Givogri, Mark S Sands, Ernesto R Bongarzone