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

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

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

http://www.wikidata.org/entity/Q34843808

about

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?Intrabodies as neuroprotective therapeutics Gene therapy in an era of emerging treatment options for hemophilia B Scalable downstream strategies for purification of recombinant adeno- associated virus vectors in light of the properties.Limitations of encapsidation of recombinant self-complementary adeno-associated viral genomes in different serotype capsids and their quantitation Progress towards gene therapy for haemophilia B.Empty Virions In AAV8 Vector Preparations Reduce Transduction Efficiency And May Cause Total Viral Particle Dose-Limiting Side-Effects Long-term safety and efficacy of factor IX gene therapy in hemophilia B.Employing a gain-of-function factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy: preclinical evaluation supporting an ongoing adeno-associated virus clinical trial.Self-complementary adeno-associated viral vectors for gene therapy of hemophilia B: progress and challenges Adenovirus-associated virus vector-mediated gene transfer in hemophilia B Preclinical dose-finding study with a liver-tropic, recombinant AAV-2/8 vector in the mouse model of galactosialidosis.In Vivo Selection Yields AAV-B1 Capsid for Central Nervous System and Muscle Gene Therapy The impact of minimally oversized adeno-associated viral vectors encoding human factor VIII on vector potency in vivo.Preclinical evaluation of an anti-HCV miRNA cluster for treatment of HCV infection Systemic errors in quantitative polymerase chain reaction titration of self-complementary adeno-associated viral vectors and improved alternative methods.Advanced Characterization of DNA Molecules in rAAV Vector Preparations by Single-stranded Virus Next-generation Sequencing.A concept of eliminating nonhomologous recombination for scalable and safe AAV vector generation for human gene therapy Chaperone-mediated gene therapy with recombinant AAV-PPCA in a new mouse model of type I sialidosis Production and purification of high-titer foamy virus vector for the treatment of leukocyte adhesion deficiency.Purification of baculovirus vectors using heparin affinity chromatography.Syngeneic AAV Pseudo-particles Potentiate Gene Transduction of AAV Vectors.Advancements in adeno-associated viral gene therapy approaches: exploring a new horizon.Engineered antibody therapies to counteract mutant huntingtin and related toxic intracellular proteins Manufacturing of recombinant adeno-associated viral vectors: new technologies are welcome Obstacles and future of gene therapy for hemophilia.New approaches to gene and cell therapy for hemophilia.Our journey to successful gene therapy for hemophilia B.Characteristics of Minimally Oversized Adeno-Associated Virus Vectors Encoding Human Factor VIII Generated Using Producer Cell Lines and Triple Transfection.AAV-ID: A Rapid and Robust Assay for Batch-to-Batch Consistency Evaluation of AAV Preparations.Absolute determination of single-stranded and self-complementary adeno-associated viral vector genome titers by droplet digital PCR.DNA Minicircle Technology Improves Purity of Adeno-associated Viral Vector Preparations.Stability and compatibility of recombinant adeno-associated virus under conditions commonly encountered in human gene therapy trials.Manufacturing of recombinant adeno-associated viruses using mammalian expression platforms.Analysis of particle content of recombinant adeno-associated virus serotype 8 vectors by ion-exchange chromatography.The Cellular Processing Capacity Limits the Amounts of Chimeric U7 snRNA Available for Antisense Delivery.Distribution of AAV8 particles in cell lysates and culture media changes with time and is dependent on the recombinant vector

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

http://www.wikidata.org/entity/Q34843808

description

2011 nî lūn-bûn

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2011 թուականի Մայիսին հրատարակուած գիտական յօդուած

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2011 թվականի մայիսին հրատարակված գիտական հոդված

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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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Good manufacturing practice pr ...... a hemophilia B clinical trial

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Good manufacturing practice pr ...... a hemophilia B clinical trial

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Human Gene Therapy

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Good manufacturing practice pr ...... a hemophilia B clinical trial

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Amit C Nathwani

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Andrew M Davidoff

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Arthur W Nienhuis

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David M Tillman

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Gael Carney

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James A Allay

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Jenny H McIntosh

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John T Gray

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Paolo Fagone

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Rob Clark

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P2860

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

Large-scale adeno-associated viral vector production using a herpesvirus-based system enables manufacturing for clinical studies

Safety and efficacy of gene transfer for Leber's congenital amaurosis

Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis

Effect of gene therapy on visual function in Leber's congenital amaurosis

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

Producing recombinant adeno-associated virus in foster cells: overcoming production limitations using a baculovirus-insect cell expression strategy.

Midbrain injection of recombinant adeno-associated virus encoding rat glial cell line-derived neurotrophic factor protects nigral neurons in a progressive 6-hydroxydopamine-induced degeneration model of Parkinson's disease in rats.

Repeat transduction in the mouse lung by using adeno-associated virus vectors with different serotypes

Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo.

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595-604

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scholarly article

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10.1089/HUM.2010.202

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5

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22

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21410419

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3081439

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