Výsledky vyhledávání

Akademický článek

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.

Autor: Laoharawee K; Center for Genome Engineering, Department of Genetics Cell Biology and Development, 6-160 Jackson Hall, 321 Church St. SE, Minneapolis, MN 55455, USA., Podetz-Pedersen KM; Center for Genome Engineering, Department of Genetics Cell Biology and Development, 6-160 Jackson Hall, 321 Church St. SE, Minneapolis, MN 55455, USA., Nguyen TT; Center for Genome Engineering, Department of Genetics Cell Biology and Development, 6-160 Jackson Hall, 321 Church St. SE, Minneapolis, MN 55455, USA., Singh SM; Center for Genome Engineering, Department of Genetics Cell Biology and Development, 6-160 Jackson Hall, 321 Church St. SE, Minneapolis, MN 55455, USA., Smith MC; Center for Genome Engineering, Department of Genetics Cell Biology and Development, 6-160 Jackson Hall, 321 Church St. SE, Minneapolis, MN 55455, USA., Belur LR; Center for Genome Engineering, Department of Genetics Cell Biology and Development, 6-160 Jackson Hall, 321 Church St. SE, Minneapolis, MN 55455, USA., Low WC; Department of Neurosurgery; University of Minnesota, Minneapolis, USA., Kozarsky KF; REGENXBIO Inc., Rockville, MD, USA., McIvor RS; Center for Genome Engineering, Department of Genetics Cell Biology and Development, 6-160 Jackson Hall, 321 Church St. SE, Minneapolis, MN 55455, USA.

Publikováno v: Molecular genetics and metabolism reports [Mol Genet Metab Rep] 2023 Jan 20; Vol. 34, pp. 100956. Date of Electronic Publication: 2023 Jan 20 (Print Publication: 2023).

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Akademický článek

Comparative Effectiveness of Intracerebroventricular, Intrathecal, and Intranasal Routes of AAV9 Vector Administration for Genetic Therapy of Neurologic Disease in Murine Mucopolysaccharidosis Type I.

Autor: Belur LR; Department of Genetics, Cell Biology and Development, Center for Genome Engineering, University of Minnesota, Minneapolis, MN, United States., Romero M; Department of Genetics, Cell Biology and Development, Center for Genome Engineering, University of Minnesota, Minneapolis, MN, United States., Lee J; Department of Genetics, Cell Biology and Development, Center for Genome Engineering, University of Minnesota, Minneapolis, MN, United States., Podetz-Pedersen KM; Department of Genetics, Cell Biology and Development, Center for Genome Engineering, University of Minnesota, Minneapolis, MN, United States., Nan Z; Department of Neurosurgery and Graduate Program in Neuroscience, University of Minnesota, Minneapolis, MN, United States., Riedl MS; Department of Neuroscience, University of Minnesota, Minneapolis, MN, United States., Vulchanova L; Department of Neuroscience, University of Minnesota, Minneapolis, MN, United States., Kitto KF; Department of Pharmaceutics, University of Minnesota, Minneapolis, MN, United States., Fairbanks CA; Department of Pharmaceutics, University of Minnesota, Minneapolis, MN, United States., Kozarsky KF; REGENXBIO Inc., Rockville, MD, United States., Orchard PJ; Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota, Minneapolis, MN, United States., Frey WH 2nd; HealthPartners Neurosciences, Regions Hospital, St. Paul, MN, United States., Low WC; Department of Neurosurgery and Graduate Program in Neuroscience, University of Minnesota, Minneapolis, MN, United States., McIvor RS; Department of Genetics, Cell Biology and Development, Center for Genome Engineering, University of Minnesota, Minneapolis, MN, United States.

Publikováno v: Frontiers in molecular neuroscience [Front Mol Neurosci] 2021 May 10; Vol. 14, pp. 618360. Date of Electronic Publication: 2021 May 10 (Print Publication: 2021).

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Akademický článek

Intravenous delivery for treatment of mucopolysaccharidosis type I: A comparison of AAV serotypes 9 and rh10.

Autor: Belur LR; Center for Genome Engineering, Department of Genetics, Cell Biology and Development, University of Minnesota, 6-160 Jackson Hall, Church St. S. E, Minneapolis, MN 55455, USA., Podetz-Pedersen KM; Center for Genome Engineering, Department of Genetics, Cell Biology and Development, University of Minnesota, 6-160 Jackson Hall, Church St. S. E, Minneapolis, MN 55455, USA., Tran TA; Center for Genome Engineering, Department of Genetics, Cell Biology and Development, University of Minnesota, 6-160 Jackson Hall, Church St. S. E, Minneapolis, MN 55455, USA., Mesick JA; Center for Genome Engineering, Department of Genetics, Cell Biology and Development, University of Minnesota, 6-160 Jackson Hall, Church St. S. E, Minneapolis, MN 55455, USA., Singh NM; Center for Genome Engineering, Department of Genetics, Cell Biology and Development, University of Minnesota, 6-160 Jackson Hall, Church St. S. E, Minneapolis, MN 55455, USA., Riedl M; Department of Neuroscience, University of Minnesota, 6-145 Jackson Hall, Church St. S.E, Minneapolis, MN 55455, USA., Vulchanova L; Department of Neuroscience, University of Minnesota, 6-145 Jackson Hall, Church St. S.E, Minneapolis, MN 55455, USA., Kozarsky KF; REGENXBIO Inc., 9600 Blackwell Road, Suite 210, Rockville, MD 20850, USA., McIvor RS; Center for Genome Engineering, Department of Genetics, Cell Biology and Development, University of Minnesota, 6-160 Jackson Hall, Church St. S. E, Minneapolis, MN 55455, USA.

Publikováno v: Molecular genetics and metabolism reports [Mol Genet Metab Rep] 2020 May 20; Vol. 24, pp. 100604. Date of Electronic Publication: 2020 May 20 (Print Publication: 2020).

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Akademický článek

A single dose of recombinant adenoviral vector rabies vaccine expressing two copies of glycoprotein protects mice from lethal virus challenge.

Autor: Qunlong Li¹, Hongzhuan He¹, Yuxi Zhou¹, Jihong Wang¹, Huan Chen¹, Hui Liu¹ liuhui@kangh.com

Publikováno v: Journal of Infection in Developing Countries. Aug2024, Vol. 18 Issue 8, p1281-1290. 10p.

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Akademický článek

Prevention of Neurocognitive Deficiency in Mucopolysaccharidosis Type II Mice by Central Nervous System-Directed, AAV9-Mediated Iduronate Sulfatase Gene Transfer.

Autor: Laoharawee K; 1 Center for Genome Engineering, Department of Genetics Cell Biology and Development, University of Minnesota , Minneapolis., Podetz-Pedersen KM; 1 Center for Genome Engineering, Department of Genetics Cell Biology and Development, University of Minnesota , Minneapolis., Nguyen TT; 1 Center for Genome Engineering, Department of Genetics Cell Biology and Development, University of Minnesota , Minneapolis., Evenstar LB; 1 Center for Genome Engineering, Department of Genetics Cell Biology and Development, University of Minnesota , Minneapolis., Kitto KF; 2 Department of Neuroscience and College of Pharmacy, University of Minnesota , Minneapolis., Nan Z; 3 Department of Neurosurgery, University of Minnesota , Minneapolis., Fairbanks CA; 2 Department of Neuroscience and College of Pharmacy, University of Minnesota , Minneapolis., Low WC; 3 Department of Neurosurgery, University of Minnesota , Minneapolis., Kozarsky KF; 4 REGENXBIO, Inc. , Rockville, Maryland., McIvor RS; 1 Center for Genome Engineering, Department of Genetics Cell Biology and Development, University of Minnesota , Minneapolis.

Publikováno v: Human gene therapy [Hum Gene Ther] 2017 Aug; Vol. 28 (8), pp. 626-638. Date of Electronic Publication: 2017 May 05.

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Akademický článek

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.

Autor: Belur LR; 1 Center for Genome Engineering, Department of Genetics, Cell Biology and Development, University of Minnesota , Minneapolis., Temme A; 1 Center for Genome Engineering, Department of Genetics, Cell Biology and Development, University of Minnesota , Minneapolis., Podetz-Pedersen KM; 1 Center for Genome Engineering, Department of Genetics, Cell Biology and Development, University of Minnesota , Minneapolis., Riedl M; 2 Department of Neuroscience, University of Minnesota , Minneapolis., Vulchanova L; 2 Department of Neuroscience, University of Minnesota , Minneapolis., Robinson N; 3 Department of Research Animal Resources, University of Minnesota , Minneapolis., Hanson LR; 4 HealthPartners Neurosciences, Regions Hospital , St. Paul, Minneapolis., Kozarsky KF; 5 REGENXBIO, Inc. , Rockville, Maryland., Orchard PJ; 6 Program in Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota , Minneapolis., Frey WH 2nd; 4 HealthPartners Neurosciences, Regions Hospital , St. Paul, Minneapolis., Low WC; 7 Department of Neurosurgery and Graduate Program in Neuroscience, University of Minnesota , Minneapolis., McIvor RS; 1 Center for Genome Engineering, Department of Genetics, Cell Biology and Development, University of Minnesota , Minneapolis.

Publikováno v: Human gene therapy [Hum Gene Ther] 2017 Jul; Vol. 28 (7), pp. 576-587. Date of Electronic Publication: 2017 Apr 20.

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