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Non-invasive intravenous administration of AAV9 transducing iduronate sulfatase leads to global metabolic correction and prevention of neurologic deficits in a mouse model of Hunter syndrome.
Laoharawee K, Podetz-Pedersen KM, Nguyen TT, Singh SM, Smith MC, Belur LR, Low WC, Kozarsky KF, McIvor RS. Laoharawee K, et al. Among authors: podetz pedersen km. Mol Genet Metab Rep. 2023 Jan 20;34:100956. doi: 10.1016/j.ymgmr.2023.100956. eCollection 2023 Mar. Mol Genet Metab Rep. 2023. PMID: 36704405 Free PMC article.
Intranasal Adeno-Associated Virus Mediated Gene Delivery and Expression of Human Iduronidase in the Central Nervous System: A Noninvasive and Effective Approach for Prevention of Neurologic Disease in Mucopolysaccharidosis Type I.
Belur LR, Temme A, Podetz-Pedersen KM, Riedl M, Vulchanova L, Robinson N, Hanson LR, Kozarsky KF, Orchard PJ, Frey WH 2nd, Low WC, McIvor RS. Belur LR, et al. Among authors: podetz pedersen km. Hum Gene Ther. 2017 Jul;28(7):576-587. doi: 10.1089/hum.2017.187. Epub 2017 Apr 20. Hum Gene Ther. 2017. PMID: 28462595 Free PMC article.
Prevention of Neurocognitive Deficiency in Mucopolysaccharidosis Type II Mice by Central Nervous System-Directed, AAV9-Mediated Iduronate Sulfatase Gene Transfer.
Laoharawee K, Podetz-Pedersen KM, Nguyen TT, Evenstar LB, Kitto KF, Nan Z, Fairbanks CA, Low WC, Kozarsky KF, McIvor RS. Laoharawee K, et al. Among authors: podetz pedersen km. Hum Gene Ther. 2017 Aug;28(8):626-638. doi: 10.1089/hum.2016.184. Epub 2017 May 5. Hum Gene Ther. 2017. PMID: 28478695
Comparative Effectiveness of Intracerebroventricular, Intrathecal, and Intranasal Routes of AAV9 Vector Administration for Genetic Therapy of Neurologic Disease in Murine Mucopolysaccharidosis Type I.
Belur LR, Romero M, Lee J, Podetz-Pedersen KM, Nan Z, Riedl MS, Vulchanova L, Kitto KF, Fairbanks CA, Kozarsky KF, Orchard PJ, Frey WH 2nd, Low WC, McIvor RS. Belur LR, et al. Among authors: podetz pedersen km. Front Mol Neurosci. 2021 May 10;14:618360. doi: 10.3389/fnmol.2021.618360. eCollection 2021. Front Mol Neurosci. 2021. PMID: 34040503 Free PMC article.
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: podetz pedersen km. 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.
24 results