Neonatal systemic gene therapy restores cardiorespiratory function in a rat model of Pompe disease
新生儿系统性基因治疗可恢复庞贝病大鼠模型的心肺功能
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doi:10.1101/2024.12.10.627800
David D Fuller, Sabhya Rana, Prajwal Thakre, Ethan Benevides, Megan Pope, Adrian G Todd, Victoria N Jensen, Lauren Vaught, Denise Cloutier, Roberto A Ribas, Reece C Larson, Matthew S Gentry, Ramon C Sun, Vijay Chandran, Manuela Corti, Darin J Falk, Barry J Byrne
