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Comparative dose effectiveness of intravenous and intrathecal AAV9.CB7.hIDS, RGX-121, in mucopolysaccharidosis type II mice.
Smith MC, Belur LR, Karlen AD, Erlanson O, Furcich J, Lund TC, Seelig D, Kitto KF, Fairbanks CA, Kim KH, Buss N, McIvor RS. Smith MC, et al. Among authors: mcivor rs. Mol Ther Methods Clin Dev. 2024 Jan 30;32(1):101201. doi: 10.1016/j.omtm.2024.101201. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38374962 Free PMC article.
Prolonged expression of a lysosomal enzyme in mouse liver after Sleeping Beauty transposon-mediated gene delivery: implications for non-viral gene therapy of mucopolysaccharidoses.
Aronovich EL, Bell JB, Belur LR, Gunther R, Koniar B, Erickson DC, Schachern PA, Matise I, McIvor RS, Whitley CB, Hackett PB. Aronovich EL, et al. Among authors: mcivor rs. J Gene Med. 2007 May;9(5):403-15. doi: 10.1002/jgm.1028. J Gene Med. 2007. PMID: 17407189 Free PMC article.
156 results