Efficacy and safety of a SOD1-targeting artificial miRNA delivered by AAV9 in mice are impacted by miRNA scaffold selection.
Chen SK, Hawley ZCE, Zavodszky MI, Hana S, Ferretti D, Grubor B, Hawes M, Xu S, Hamann S, Marsh G, Cullen P, Challa R, Carlile TM, Zhang H, Lee WH, Peralta A, Clarner P, Wei C, Koszka K, Gao F, Lo SC.
Chen SK, et al. Among authors: xu s.
Mol Ther Nucleic Acids. 2023 Oct 16;34:102057. doi: 10.1016/j.omtn.2023.102057. eCollection 2023 Dec 12.
Mol Ther Nucleic Acids. 2023.
PMID: 37928442
Free PMC article.