Immunomodulatory gene therapy prevents antibody formation and lethal hypersensitivity reactions in murine pompe disease. - Wikidata - awgldk - TriplyDB

Immunomodulatory gene therapy prevents antibody formation and lethal hypersensitivity reactions in murine pompe disease.

Immunomodulatory gene therapy prevents antibody formation and lethal hypersensitivity reactions in murine pompe disease.

http://www.wikidata.org/entity/Q33637062

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Targeted approaches to induce immune tolerance for Pompe disease therapy New insights into therapeutic options for Pompe disease Immune responses and hypercoagulation in ERT for Pompe disease are mutation and rhGAA dose dependent.Oral delivery of bioencapsulated coagulation factor IX prevents inhibitor formation and fatal anaphylaxis in hemophilia B mice.Immunomodulatory gene therapy in lysosomal storage disorders AAV-mediated liver-directed gene therapy.Mapping the T helper cell response to acid α-glucosidase in Pompe mice Transient B cell depletion or improved transgene expression by codon optimization promote tolerance to factor VIII in gene therapy.The pharmacological chaperone AT2220 increases recombinant human acid α-glucosidase uptake and glycogen reduction in a mouse model of Pompe disease.Development of Gene Transfer for Induction of Antigen-specific Tolerance.Pompe disease gene therapy.Enhanced efficacy from gene therapy in Pompe disease using coreceptor blockade Assessment of toxicity and biodistribution of recombinant AAV8 vector-mediated immunomodulatory gene therapy in mice with Pompe disease.Comparative impact of AAV and enzyme replacement therapy on respiratory and cardiac function in adult Pompe mice Immunodominant liver-specific expression suppresses transgene-directed immune responses in murine pompe disease Synergistic Efficacy from Gene Therapy with Coreceptor Blockade and a β2-Agonist in Murine Pompe Disease.Adjunctive β2-agonists reverse neuromuscular involvement in murine Pompe disease.Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease.Deficiency in MyD88 Signaling Results in Decreased Antibody Responses to an Adeno-Associated Virus Vector in Murine Pompe Disease.A beta-blocker, propranolol, decreases the efficacy from enzyme replacement therapy in Pompe disease.Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates.Effective gene therapy for haemophilic mice with pathogenic factor IX antibodies.Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients.Low-dose Gene Therapy Reduces the Frequency of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage Disease.Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction.Emerging drugs for lysosomal storage diseases.The complex and evolving story of T cell activation to AAV vector-encoded transgene products.Liver gene therapy by lentiviral vectors reverses anti-factor IX pre-existing immunity in haemophilic mice Teaching tolerance: New approaches to enzyme replacement therapy for Pompe disease The role of immune tolerance induction in restoration of the efficacy of ERT in Pompe disease.Prevention and Reversal of Antibody Responses Against Factor IX in Gene Therapy for Hemophilia B.Enhanced delivery of α-glucosidase for Pompe disease by ICAM-1-targeted nanocarriers: comparative performance of a strategy for three distinct lysosomal storage disorders.Hepatic AAV gene transfer and the immune system: friends or foes?Administration of anti-CD3 antibodies modulates the immune response to an infusion of α-glucosidase in mice Nonredundant roles of IL-10 and TGF-β in suppression of immune responses to hepatic AAV-factor IX gene transfer Minimum requirement of donor cells to reduce the glycolipid storage following bone marrow transplantation in a murine model of Fabry disease.Gene Therapy for Hemophilia: Progress to Date.Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase.Immunomodulatory, liver depot gene therapy for Pompe disease.The new era of Pompe disease: advances in the detection, understanding of the phenotypic spectrum, pathophysiology, and management.

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P2860

Immunomodulatory gene therapy prevents antibody formation and lethal hypersensitivity reactions in murine pompe disease.

http://www.wikidata.org/entity/Q33637062

description

2009 nî lūn-bûn

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2009 թուականի Օգոստոսին հրատարակուած գիտական յօդուած

@hyw

2009 թվականի օգոստոսին հրատարակված գիտական հոդված

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2009年の論文

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2009年論文

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2009年論文

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2009年論文

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2009年論文

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2009年論文

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2009年论文

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Immunomodulatory gene therapy ...... tions in murine pompe disease.

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Immunomodulatory gene therapy ...... tions in murine pompe disease.

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Immunomodulatory gene therapy ...... tions in murine pompe disease.

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Immunomodulatory gene therapy ...... tions in murine pompe disease.

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Molecular Therapy

P1476

Immunomodulatory gene therapy ...... tions in murine pompe disease.

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P2093

Amy C Hobeika

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Andrew Bird

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Baodong Sun

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Dwight D Koeberl

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Haoyue Zhang

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Michael D Kulis

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Priya S Kishnani

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Sarah P Young

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Songtao Li

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Timothy M Clay

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P2860

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease

Targeted disruption of the acid alpha-glucosidase gene in mice causes an illness with critical features of both infantile and adult human glycogen storage disease type II

Chinese hamster ovary cell-derived recombinant human acid alpha-glucosidase in infantile-onset Pompe disease.

Recombinant human acid alpha-glucosidase enzyme therapy for infantile glycogen storage disease type II: results of a phase I/II clinical trial.

Enzyme replacement therapy in Fabry disease: a randomized controlled trial.

Immune tolerance after long-term enzyme-replacement therapy among patients who have mucopolysaccharidosis I.

Systemic correction of the muscle disorder glycogen storage disease type II after hepatic targeting of a modified adenovirus vector encoding human acid-alpha-glucosidase

Sustained correction of bleeding disorder in hemophilia B mice by gene therapy.

Enhanced response to enzyme replacement therapy in Pompe disease after the induction of immune tolerance.

P304

353-360

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scholarly article

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10.1038/MT.2009.195

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2

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18

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26751286

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19690517

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2818301

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