Intrathecal or intravenous AAV9-IDUA/RGX-111 at minimal effective dose prevents cardiac, skeletal and neurologic manifestations of murine MPS I.
鞘内或静脉注射最小有效剂量的 AAV9-IDUA/RGX-111 可预防小鼠 MPS I 的心脏、骨骼和神经系统症状
期刊:Molecular Therapy-Methods & Clinical Development
影响因子:4.7
doi:10.1016/j.omtm.2024.101369
Belur Lalitha R, Huber Avery K, Mantone Hillary, Robertson Mason, Smith Miles C, Karlen Andrea D, Kitto Kelley F, Ou Li, Whitley Chester B, Braunlin Elizabeth, Furcich Justin, Lund Troy C, Seelig Davis, Fairbanks Carolyn A, Buss Nicholas, Kim Kwi Hye, McIvor R Scott
