Assessment of Safety and Biodistribution of AAVrh.10hCLN2 Following Intracisternal Administration in Nonhuman Primates for the Treatment of CLN2 Batten Disease
在非人灵长类动物中,通过脑池内给药评估 AAVrh.10hCLN2 治疗 CLN2 巴顿病的安全性和生物分布
期刊:Human Gene Therapy
影响因子:4
doi:10.1089/hum.2023.067
De, Bishnu P; Rosenberg, Jonathan B; Selvan, Nithya; Wilson, Isabelle; Yusufzai, Nadir; Greco, Alessandria; Kaminsky, Stephen M; Heier, Linda A; Ricart Arbona, Rodolfo J; Miranda, Ileana C; Monette, Sebastien; Nair, Anju; Khanna, Richie; Crystal, Ronald G; Sondhi, Dolan