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Phenotypic Correction of Murine Mucopolysaccharidosis Type II by Engraftment of Ex Vivo Lentiviral Vector-Transduced Hematopoietic Stem and Progenitor Cells.
Smith MC, Belur LR, Karlen AD, Erlanson O, Podetz-Pedersen KM, McKenzie J, Detellis J, Gagnidze K, Parsons G, Robinson N, Labarre S, Shah S, Furcich J, Lund TC, Tsai HC, McIvor RS, Bonner M. Smith MC, et al. Among authors: furcich j. Hum Gene Ther. 2022 Dec;33(23-24):1279-1292. doi: 10.1089/hum.2022.141. Epub 2022 Nov 23. Hum Gene Ther. 2022. PMID: 36226412 Free PMC article.
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: furcich j. 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 Free PMC article.
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: furcich j. 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.