AAV-based neonatal gene therapy for hemophilia A: long-term correction and avoidance of immune responses in mice
about
Gene therapy for hemophiliaPre-clinical efficacy and dosing of an AAV8 vector expressing human methylmalonyl-CoA mutase in a murine model of methylmalonic acidemia (MMA)Rescue of cardiomyopathy through U7snRNA-mediated exon skipping in Mybpc3-targeted knock-in miceOptimization of scAAVIL-1ra In Vitro and In Vivo to Deliver High Levels of Therapeutic Protein for Treatment of Osteoarthritis.Development of operational immunologic tolerance with neonatal gene transfer in nonhuman primates: preliminary studies.Minimal ureagenesis is necessary for survival in the murine model of hyperargininemia treated by AAV-based gene therapy.Myocyte-mediated arginase expression controls hyperargininemia but not hyperammonemia in arginase-deficient mice.Evaluation of transduction properties of an adenovirus vector in neonatal mice.Augmentation of transgene-encoded protein after neonatal injection of adeno-associated virus improves hepatic copy number without immune responses.Long-term survival of the juvenile lethal arginase-deficient mouse with AAV gene therapy.Generation of an optimized lentiviral vector encoding a high-expression factor VIII transgene for gene therapy of hemophilia AExpressing Transgenes That Exceed the Packaging Capacity of Adeno-Associated Virus Capsids.AAV-based gene therapy prevents neuropathology and results in normal cognitive development in the hyperargininemic mouse.Rescue of the Functional Alterations of Motor Cortical Circuits in Arginase Deficiency by Neonatal Gene Therapy.Pseudotyped murine leukemia virus for schistosome transgenesis: approaches, methods and perspectives.Enhanced Proteolytic Processing of Recombinant Human Coagulation Factor VIII B-Domain Variants by Recombinant Furins.Neonatal Gene Therapy for Hemophilia B by a Novel Adenovirus Vector Showing Reduced Leaky Expression of Viral Genes.Optimized AAV rh.10 Vectors That Partially Evade Neutralizing Antibodies during Hepatic Gene Transfer.Improved adeno-associated virus (AAV) serotype 1 and 5 vectors for gene therapy.Innovative Approaches for Immune Tolerance to Factor VIII in the Treatment of Hemophilia A.Intramuscular Adeno-Associated Virus-Mediated Expression of Monoclonal Antibodies Provides 100% Protection Against Ebola Virus Infection in Mice.
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P2860
AAV-based neonatal gene therapy for hemophilia A: long-term correction and avoidance of immune responses in mice
description
2012 nî lūn-bûn
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2012年の論文
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2012年論文
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2012年論文
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2012年論文
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2012年論文
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2012年論文
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2012年论文
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name
AAV-based neonatal gene therap ...... ce of immune responses in mice
@ast
AAV-based neonatal gene therap ...... ce of immune responses in mice
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type
label
AAV-based neonatal gene therap ...... ce of immune responses in mice
@ast
AAV-based neonatal gene therap ...... ce of immune responses in mice
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prefLabel
AAV-based neonatal gene therap ...... ce of immune responses in mice
@ast
AAV-based neonatal gene therap ...... ce of immune responses in mice
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AAV-based neonatal gene therap ...... ce of immune responses in mice
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10.1038/GT.2011.200
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2012-01-12T00:00:00Z
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1028365513