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An in vitro and in vivo efficacy evaluation of gene therapy candidate SBT101 in mouse models of adrenomyeloneuropathy and in NHPs.
Vasireddy V, Maguire CA, Anderson DW, Ng C, Gong Y, Eichler F, Fourcade S, Guilera C, Onieva A, Sanchez A, Leal-Julià M, Verdés S, Dijkstra IME, Kemp S, Park H, Lutz T, Clark SW, Bosch A, Pujol A, Kozarsky K. Vasireddy V, et al. Among authors: kozarsky k. Mol Ther Methods Clin Dev. 2024 Oct 9;32(4):101354. doi: 10.1016/j.omtm.2024.101354. eCollection 2024 Dec 12. Mol Ther Methods Clin Dev. 2024. PMID: 39524975 Free PMC article.
In vivo selection in non-human primates identifies AAV capsids for on-target CSF delivery to spinal cord.
Hanlon KS, Cheng M, Ferrer RM, Ryu JR, Lee B, De La Cruz D, Patel N, Espinoza P, Santoscoy MC, Gong Y, Ng C, Nguyen DM, Nammour J, Clark SW, Heine VM, Sun W, Kozarsky K, Maguire CA. Hanlon KS, et al. Among authors: kozarsky k. Mol Ther. 2024 Aug 7;32(8):2584-2603. doi: 10.1016/j.ymthe.2024.05.040. Epub 2024 Jun 5. Mol Ther. 2024. PMID: 38845196 Free PMC article.
In vivo selection in non-human primates identifies superior AAV capsids for on-target CSF delivery to spinal cord.
Hanlon KS, Cheng M, De La Cruz D, Patel N, Santoscoy MC, Gong Y, Ng C, Nguyen DM, Nammour J, Clark SW, Kozarsky K, Maguire CA. Hanlon KS, et al. Among authors: kozarsky k. bioRxiv [Preprint]. 2023 Sep 13:2023.09.13.557506. doi: 10.1101/2023.09.13.557506. bioRxiv. 2023. Update in: Mol Ther. 2024 Aug 7;32(8):2584-2603. doi: 10.1016/j.ymthe.2024.05.040 PMID: 37745398 Free PMC article. Updated. Preprint.
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: kozarsky kf. 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.
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: kozarsky kf. 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.
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: kozarsky kf. 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.
67 results