Long-term safety and efficacy of factor IX gene therapy in hemophilia B. - Wikidata - awgldk - TriplyDB

Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

http://www.wikidata.org/entity/Q34783922

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Clinical development of gene therapy: results and lessons from recent successes Molecular Imaging in Genetic Medicine Manufacturing of recombinant adeno-associated viral vectors for clinical trials Gene therapy: progress and predictions Emerging and future therapies for hemophilia Cell-based therapy technology classifications and translational challenges Engineering humoral immunity as prophylaxis or therapy AAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form of Batten disease.CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome)Genome-editing technologies for gene correction of hemophilia Once-weekly prophylactic dosing of recombinant factor IX improves adherence in hemophilia B The CD8 T-cell response during tolerance induction in liver transplantation Hemophilia A gene therapy via intraosseous delivery of factor VIII-lentiviral vectors E Pluribus Unum: 50 Years of Research, Millions of Viruses, and One Goal--Tailored Acceleration of AAV Evolution Gene therapy in an era of emerging treatment options for hemophilia B Gene Therapy for Metabolic Diseases The current state of adverse event reporting in hemophilia.Ultrasound-targeted hepatic delivery of factor IX in hemophiliac mice.Engineering antibody-like inhibitors to prevent and treat HIV-1 infection.Translational data from adeno-associated virus-mediated gene therapy of hemophilia B in dogs Early clinical data raise the bar for hemophilia gene therapies.ADAMTS13 and anti-ADAMTS13 autoantibodies in thrombotic thrombocytopenic purpura - current perspectives and new treatment strategies.Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models Low-Dose Gene Therapy for Murine PKU Using Episomal Naked DNA Vectors Expressing PAH from Its Endogenous Liver Promoter Concise Review: The Potential Use of Intestinal Stem Cells to Treat Patients with Intestinal Failure.Many factor VIII products available in the treatment of hemophilia A: an embarrassment of riches?Targeted gene knock-in by homology-directed genome editing using Cas9 ribonucleoprotein and AAV donor delivery.Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects.Gene Therapy 2017: Progress and Future Directions.AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice.Profile of efraloctocog alfa and its potential in the treatment of hemophilia A.Hitting the target without pulling the trigger Cystic Fibrosis Gene Therapy in the UK and Elsewhere.Vectored antibody gene delivery for the prevention or treatment of HIV infection.Hemophilia B: molecular pathogenesis and mutation analysis.Pathogenic mechanisms and the prospect of gene therapy for choroideremia.Successful Phenotype Improvement following Gene Therapy for Severe Hemophilia A in Privately Owned Dogs.Recombinant AAV Vectors for Enhanced Expression of Authentic IgG.In vivo genome editing of the albumin locus as a platform for protein replacement therapy Long-term rescue of cone photoreceptor degeneration in retinitis pigmentosa 2 (RP2)-knockout mice by gene replacement therapy.

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P2860

Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

http://www.wikidata.org/entity/Q34783922

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2014 nî lūn-bûn

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2014 թուականի Նոյեմբերին հրատարակուած գիտական յօդուած

@hyw

2014 թվականի նոյեմբերին հրատարակված գիտական հոդված

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2014年の論文

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2014年論文

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2014年論文

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2014年論文

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2014年論文

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2014年論文

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2014年论文

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name

Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

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Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

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Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

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Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

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Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

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Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

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Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

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Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

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Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

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The New England Journal of Medicine

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Long-term safety and efficacy of factor IX gene therapy in hemophilia B

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Amit C Nathwani

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Andrew M Davidoff

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Anne Riddell

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Arthur W Nienhuis

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Catherine Y C Ng

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Cecilia Rosales

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Christopher L Morton

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David Bevan

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Deepak Raj

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

Deokumar Srivastava

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

P2860

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

Analysis of low frequency bleeding data: the association of joint bleeds according to baseline FVIII activity levels.

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

Good manufacturing practice production of self-complementary serotype 8 adeno-associated viral vector for a hemophilia B clinical trial

Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins.

Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates

Adenovirus-associated virus vector-mediated gene transfer in hemophilia B

Epidemiology of coagulation disorders.

Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver

Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus.

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1994-2004

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scholarly article

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10.1056/NEJMOA1407309

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P433

21

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371

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Marco Della Peruta

Pratima Chowdary

Katherine A High

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2014-11-01T00:00:00Z

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25409372

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Coagulation factor IX

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4278802

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