Intrathecal or intravenous AAV9-IDUA/RGX-111 at minimal effective dose prevents cardiac, skeletal and neurologic manifestations of murine MPS I.
Belur LR, Huber AK, Mantone H, Robertson M, Smith MC, Karlen AD, Kitto KF, Ou L, Whitley CB, Braunlin E, Furcich J, Lund TC, Seelig D, Fairbanks CA, Buss N, Kim KH, McIvor RS.
Belur LR, et al. Among authors: smith mc.
Mol Ther Methods Clin Dev. 2024 Nov 4;32(4):101369. doi: 10.1016/j.omtm.2024.101369. eCollection 2024 Dec 12.
Mol Ther Methods Clin Dev. 2024.
PMID: 39687731
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