AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. - Wikidata - wikimedia - TriplyDB

AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.

AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.

http://www.wikidata.org/entity/Q34168942

about

Role of the vector genome and underlying factor IX mutation in immune responses to AAV gene therapy for hemophilia B Gene therapy for severe combined immunodeficiency: are we there yet?Clinical development of gene therapy: results and lessons from recent successes Manufacturing of recombinant adeno-associated viral vectors for clinical trials The interplay of post-translational modification and gene therapy Developments in the treatment of hemophilia B: focus on emerging gene therapy The gene therapy journey for hemophilia: are we there yet?Parkinson's disease gene therapy: success by design meets failure by efficacy Animal models of hemophilia Adeno-associated virus-mediated cancer gene therapy: current status Adeno-associated virus: from defective virus to effective vector Current animal models of hemophilia: the state of the art Gene therapy for hemophilia Gene therapy in an era of emerging treatment options for hemophilia B Long-term rescue of a lethal inherited disease by adeno-associated virus-mediated gene transfer in a mouse model of molybdenum-cofactor deficiency Development and utility of an internal threshold control (ITC) real-time PCR assay for exogenous DNA detection Proteasome inhibition is partially effective in attenuating pre-existing immunity against recombinant adeno-associated viral vectors Development of Patient-specific AAV Vectors After Neutralizing Antibody Selection for Enhanced Muscle Gene Transfer Lessons Learned from Animal Models of Inherited Bleeding Disorders.Treatment of diabetes and long-term survival after insulin and glucokinase gene therapy.Translational data from adeno-associated virus-mediated gene therapy of hemophilia B in dogs Directed evolution of adeno-associated virus yields enhanced gene delivery vectors.A look to future directions in gene therapy research for monogenic diseases.Application of a haematopoetic progenitor cell-targeted adeno-associated viral (AAV) vector established by selection of an AAV random peptide library on a leukaemia cell line.Directed evolution of a novel adeno-associated virus (AAV) vector that crosses the seizure-compromised blood-brain barrier (BBB).AAV's anatomy: roadmap for optimizing vectors for translational success Gene expression in lung and liver after intravenous infusion of polyethylenimine complexes of Sleeping Beauty transposons.Host and vector-dependent effects on the risk of germline transmission of AAV vectors.Strategies to modulate immune responses: a new frontier for gene therapy Comparative analysis of virus-host interactions caused by a virulent and an attenuated duck hepatitis A virus genotype 1 Comparison of adeno-associated virus pseudotype 1, 2, and 8 vectors administered by intramuscular injection in the treatment of murine phenylketonuria Optimized adeno-associated virus (AAV)-protein phosphatase-5 helper viruses for efficient liver transduction by single-stranded AAV vectors: therapeutic expression of factor IX at reduced vector doses.Progress towards gene therapy for haemophilia B.Hoechst increases adeno-associated virus-mediated transgene expression in airway epithelia by inducing the cytomegalovirus promoter Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects.The potential of adeno-associated viral vectors for gene delivery to muscle tissue.Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B.Cell-Mediated Immunity to AAV Vectors, Evolving Concepts and Potential Solutions.Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs.Factor IX ectopically expressed in platelets can be stored in alpha-granules and corrects the phenotype of hemophilia B mice.

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P2860

AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.

http://www.wikidata.org/entity/Q34168942

description

2002 nî lūn-bûn

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2002 թուականի Դեկտեմբերին հրատարակուած գիտական յօդուած

@hyw

2002 թվականի դեկտեմբերին հրատարակված գիտական հոդված

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2002年の論文

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2002年学术文章

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2002年学术文章

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2002年学术文章

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2002年学术文章

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2002年学术文章

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2002年學術文章

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name

AAV-mediated factor IX gene tr ...... ents with severe hemophilia B.

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AAV-mediated factor IX gene tr ...... ents with severe hemophilia B.

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AAV-mediated factor IX gene tr ...... ents with severe hemophilia B.

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type

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AAV-mediated factor IX gene tr ...... ents with severe hemophilia B.

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AAV-mediated factor IX gene tr ...... ents with severe hemophilia B.

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AAV-mediated factor IX gene tr ...... ents with severe hemophilia B.

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prefLabel

AAV-mediated factor IX gene tr ...... ents with severe hemophilia B.

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AAV-mediated factor IX gene tr ...... ents with severe hemophilia B.

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AAV-mediated factor IX gene tr ...... ents with severe hemophilia B.

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AAV-mediated factor IX gene tr ...... ents with severe hemophilia B.

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Alan W Flake

http://www.w3.org/2001/XMLSchema#string

Amy J Chew

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Arthur Thompson

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Bertil Glader

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Ciaran Scallan

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Debra G B Leonard

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Katherine A High

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Linda B Couto

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Margaret V Ragni

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2963-2972

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scholarly article

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10.1182/BLOOD-2002-10-3296

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8

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Catherine Manno

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10960759

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12515715

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Coagulation factor IX