Prolonged expression of a lysosomal enzyme in mouse liver after Sleeping Beauty transposon-mediated gene delivery: implications for non-viral gene therapy of mucopolysaccharidoses - Wikidata - awgldk - TriplyDB

Prolonged expression of a lysosomal enzyme in mouse liver after Sleeping Beauty transposon-mediated gene delivery: implications for non-viral gene therapy of mucopolysaccharidoses

Prolonged expression of a lysosomal enzyme in mouse liver after Sleeping Beauty transposon-mediated gene delivery: implications for non-viral gene therapy of mucopolysaccharidoses

http://www.wikidata.org/entity/Q24684749

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A transposon and transposase system for human application Gene therapy for mucopolysaccharidosis Systemic correction of storage disease in MPS I NOD/SCID mice using the sleeping beauty transposon system The Sleeping Beauty transposon system: a non-viral vector for gene therapy Preferential delivery of the Sleeping Beauty transposon system to livers of mice by hydrodynamic injection Therapies for the bone in mucopolysaccharidoses A Broad Range of Dose Optima Achieve High-level, Long-term Gene Expression After Hydrodynamic Delivery of Sleeping Beauty Transposons Using Hyperactive SB100x Transposase Liver-targeted gene therapy: Approaches and challenges DNA transposon-based gene vehicles - scenes from an evolutionary drive DNA transposons: nature and applications in genomics Improved retroviral vector design results in sustained expression after adult gene therapy in mucopolysaccharidosis I mice.Gene expression in lung and liver after intravenous infusion of polyethylenimine complexes of Sleeping Beauty transposons.Gene and cell therapy for children--new medicines, new challenges?Helper-independent piggyBac plasmids for gene delivery approaches: strategies for avoiding potential genotoxic effects.A new extensively characterised conditionally immortal muscle cell-line for investigating therapeutic strategies in muscular dystrophies Translating Sleeping Beauty transposition into cellular therapies: victories and challenges.Duration of expression and activity of Sleeping Beauty transposase in mouse liver following hydrodynamic DNA delivery Herpes simplex virus/Sleeping Beauty vector-based embryonic gene transfer using the HSB5 mutant: loss of apparent transposition hyperactivity in vivo Therapies of mucopolysaccharidosis IVA (Morquio A syndrome).Cellular immune response against firefly luciferase after sleeping beauty-mediated gene transfer in vivo Inhibition of angiogenesis and suppression of colorectal cancer metastatic to the liver using the Sleeping Beauty Transposon System Minicircle DNA-based gene therapy coupled with immune modulation permits long-term expression of α-L-iduronidase in mice with mucopolysaccharidosis type I Lysosomal storage disease: gene therapy on both sides of the blood-brain barrier.Quantitative analysis of α-L-iduronidase expression in immunocompetent mice treated with the Sleeping Beauty transposon system Mesenchymal stem cells do not prevent antibody responses against human α-L-iduronidase when used to treat mucopolysaccharidosis type I.Gene therapy for neurologic manifestations of mucopolysaccharidoses.Hybrid nonviral/viral vector systems for improved piggyBac DNA transposon in vivo delivery Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I.Retargeting transposon insertions by the adeno-associated virus Rep protein.Pigs taking wing with transposons and recombinases.Predicting preferential DNA vector insertion sites: implications for functional genomics and gene therapy.A Hyperactive Transposase Promotes Persistent Gene Transfer of a piggyBac DNA Transposon Trehalose-Based Block Copolycations Promote Polyplex Stabilization for Lyophilization and in Vivo pDNA Delivery.Gene therapy for cardiovascular manifestations of lysosomal storage diseases.Targeting of the CNS in MPS-IH using a nonviral transferrin-alpha-L-iduronidase fusion gene product.Functional characterization of the Bari1 transposition system.The expanding universe of transposon technologies for gene and cell engineering.Efficacy and safety of Sleeping Beauty transposon-mediated gene transfer in preclinical animal studies.The Sleeping Beauty transposon system for clinical applications.Recent developments in transposon-mediated gene therapy.

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Prolonged expression of a lysosomal enzyme in mouse liver after Sleeping Beauty transposon-mediated gene delivery: implications for non-viral gene therapy of mucopolysaccharidoses

http://www.wikidata.org/entity/Q24684749

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2007 nî lūn-bûn

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Chester B Whitley

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David C C Erickson

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Elena L Aronovich

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Ilze Matise

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Jason B Bell

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Perry B Hackett

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R Scott McIvor

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P2860

Murine mucopolysaccharidosis type VII. Characterization of a mouse with beta-glucuronidase deficiency

Murine mucopolysaccharidosis type I: targeted disruption of the murine alpha-L-iduronidase gene

Molecular reconstruction of Sleeping Beauty, a Tc1-like transposon from fish, and its transposition in human cells

Bone marrow transplantation for globoid cell leukodystrophy, adrenoleukodystrophy, metachromatic leukodystrophy, and Hurler syndrome.

High levels of foreign gene expression in hepatocytes after tail vein injections of naked plasmid DNA.

Structure-function analysis of the inverted terminal repeats of the sleeping beauty transposon.

Gene transfer into genomes of human cells by the sleeping beauty transposon system.

Cardiac manifestations in the mouse model of mucopolysaccharidosis I.

Sleeping beauty transposon-mediated gene therapy for prolonged expression.

Progress and prospects: naked DNA gene transfer and therapy.

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