Preclinical evaluation of a clinical candidate AAV8 vector for ornithine transcarbamylase (OTC) deficiency reveals functional enzyme from each persisting vector genome.

Preclinical evaluation of a clinical candidate AAV8 vector for ornithine transcarbamylase (OTC) deficiency reveals functional enzyme from each persisting vector genome.

Item
http://www.wikidata.org/entity/Q35726773
Low-Dose Gene Therapy for Murine PKU Using Episomal Naked DNA Vectors Expressing PAH from Its Endogenous Liver PromoterNovel two-step derivation method for the synchronous analysis of inherited metabolic disorders using urine.Transient B cell depletion or improved transgene expression by codon optimization promote tolerance to factor VIII in gene therapy.Comparative Study of Liver Gene Transfer With AAV Vectors Based on Natural and Engineered AAV Capsids.Genetic Manipulation of Brown Fat Via Oral Administration of an Engineered Recombinant Adeno-associated Viral Serotype Vector.Liver-directed adeno-associated virus serotype 8 gene transfer rescues a lethal murine model of citrullinemia type 1.Stable isotopes in the diagnosis and treatment of inherited hyperammonemiaAdeno-Associated Virus Gene Therapy for Liver Disease.Alternative Start Sites Downstream of Non-Sense Mutations Drive Antigen Presentation and Tolerance Induction to C-Terminal Epitopes.AAV gene therapy corrects OTC deficiency and prevents liver fibrosis in aged OTC-knock out heterozygous mice.Optimized human factor IX expression cassettes for hepatic-directed gene therapy of hemophilia B.AAV vectors expressing LDLR gain-of-function variants demonstrate increased efficacy in mouse models of familial hypercholesterolemia.A novel mutation of the ornithine transcarbamylase gene leading to fatal hyperammonemia in a liver transplant recipient.Gene Therapy for Hemophilia: Progress to Date.Hiding in Plain Sight: A Case of Ornithine Transcarbamylase Deficiency Unmasked Post-Liver Transplantation.Targeted mRNA Therapy for Ornithine Transcarbamylase Deficiency.Nuclease-Mediated Gene Therapies for Inherited Metabolic Diseases of the Liver.
P2860

Preclinical evaluation of a clinical candidate AAV8 vector for ornithine transcarbamylase (OTC) deficiency reveals functional enzyme from each persisting vector genome.

Item
http://www.wikidata.org/entity/Q35726773
description
2011 nî lūn-bûn
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2011年の論文
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2011年論文
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2011年論文
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2011年論文
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2011年論文
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2011年論文
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2011年论文
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2011年论文
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2011年论文
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Preclinical evaluation of a cl ...... each persisting vector genome.
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Preclinical evaluation of a cl ...... each persisting vector genome.
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Preclinical evaluation of a cl ...... each persisting vector genome.
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Preclinical evaluation of a cl ...... each persisting vector genome.
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Preclinical evaluation of a cl ...... each persisting vector genome.
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Preclinical evaluation of a cl ...... each persisting vector genome.
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Preclinical evaluation of a cl ...... each persisting vector genome.
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10.1016/J.YMGME.2011.10.020
P433
P478
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