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Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations: Safety and Efficacy in 15 Children and Adults Followed Up to 3 Years

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Viral Concentration Determination Through Plaque Assays: Using Traditional and Novel Overlay Systems

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Rapid, Simple, and Versatile Manufacturing of Recombinant Adeno-Associated Viral Vectors at Scale

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α2,3 and α2,6 N-Linked Sialic Acids Facilitate Efficient Binding and Transduction by Adeno-Associated Virus Types 1 and 6

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Production of Recombinant Adeno-associated Virus Vectors Using Suspension HEK293 Cells and Continuous Harvest of Vector From the Culture Media for GMP FIX and FLT1 Clinical Vector

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Adeno-Associated Virus Type 6 (AAV6) Vectors Mediate Efficient Transduction of Airway Epithelial Cells in Mouse Lungs Compared to That of AAV2 Vectors

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Highly Purified Recombinant Adeno-Associated Virus Vectors Are Biologically Active and Free of Detectable Helper and Wild-Type Viruses

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Progress and challenges in viral vector manufacturing

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A Simplified Baculovirus-AAV Expression Vector System Coupled With One-step Affinity Purification Yields High-titer rAAV Stocks From Insect Cells

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Cell Lines for the Production of Recombinant Adeno-Associated Virus

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CpG-depleted adeno-associated virus vectors evade immune detection

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The genome of self-complementary adeno-associated viral vectors increases Toll-like receptor 9–dependent innate immune responses in the liver

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Characterization of Genome Integrity for Oversized Recombinant AAV Vector

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Quantification of adeno-associated virus particles and empty capsids by optical density measurement

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Efficient and persistent gene transfer of AAV-CFTR in maxillary sinus

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Basic Biology of Adeno-Associated Virus (AAV) Vectors Used in Gene Therapy

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Immune responses to adeno-associated virus and its recombinant vectors.

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Effects of ammonia on CHO cell growth, erythropoietin production, and glycosylation

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Safety and Biological Efficacy of an Adeno‐Associated Virus Vector–Cystic Fibrosis Transmembrane Regulator (AAV‐CFTR) in the Cystic Fibrosis Maxillary Sinus

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Transduction by adeno-associated virus vectors in the rabbit airway: efficiency, persistence, and readministration

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Manufacturing and characterizing AAV-based vectors for use in clinical studies

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Titration of AAV-2 particles via a novel capsid ELISA: packaging of genomes can limit production of recombinant AAV-2

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High-Titer Adeno-Associated Viral Vectors from a Rep/Cap Cell Line and Hybrid Shuttle Virus

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Development of formulations that enhance physical stability of viral vectors for gene therapy

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Purification of Recombinant Adeno-Associated Virus by Iodixanol Gradient Ultracentrifugation Allows Rapid and Reproducible Preparation of Vector Stocks for Gene Transfer in the Nervous System

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Scalable Purification of Adeno-Associated Virus Type 2, 4, or 5 Using Ion-Exchange Chromatography

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Absolute determination of single-stranded and self-complementary adeno-associated viral vector genome titers by droplet digital PCR

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Viral Concentration Determination Through Plaque Assays: Using Traditional and Novel Overlay Systems

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Electron cryo‐microscopy and image reconstruction of adeno‐associated virus type 2 empty capsids

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Comparative Analysis of Adeno-Associated Virus Capsid Stability and Dynamics

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Identification of factors that contribute to recombinant AAV2 particle aggregation and methods to prevent its occurrence during vector purification and formulation

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Separation of adeno-associated virus type 2 empty particles from genome containing vectors by anion-exchange column chromatography

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High-titer recombinant adeno-associated virus production utilizing a recombinant herpes simplex virus type I vector expressing AAV-2 Rep and Cap

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

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Resolving Adeno-Associated Viral Particle Diversity With Charge Detection Mass Spectrometry

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Analytical Ultracentrifugation as an Approach to Characterize Recombinant Adeno-Associated Viral Vectors

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Fast and reliable titration of recombinant adeno-associated virus type-2 using quantitative real-time PCR

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Biophysical and Ultrastructural Characterization of Adeno-Associated Virus Capsid Uncoating and Genome Release

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Adeno-Associated Virus Biology

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Transient transfection methods for clinical adeno-associated viral vector production

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Immune responses to AAV-vectors, the Glybera example from bench to bedside

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Large-Scale Adeno-Associated Viral Vector Production Using a Herpesvirus-Based System Enables Manufacturing for Clinical Studies

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Production of adeno-associated virus (AAV) serotypes by transient transfection of HEK293 cell suspension cultures for gene delivery

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Scalable Purification of Adeno-associated Virus Serotype 1 (AAV1) and AAV8 Vectors, Using Dual Ion-Exchange Adsorptive Membranes

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Evidence for Encapsidation of Prokaryotic Sequences during Recombinant Adeno-Associated Virus Production and Their in Vivo Persistence after Vector Delivery

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Current issues in adeno-associated viral vector production

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Analysis of particle content of recombinant adeno-associated virus serotype 8 vectors by ion-exchange chromatography

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Reproducible High Yields of Recombinant Adeno-Associated Virus Produced Using Invertebrate Cells in 0.02- to 200-Liter Cultures

Cecchini, S., Virág, T., & Kotin, R. M. (2011) · Human Gene Therapy · doi:10.1089/hum.2010.250 · Cited by 98 · see citing works

Systemic errors in quantitative polymerase chain reaction titration of self-complementary adeno-associated viral vectors and improved alternative methods

Fagone, P., Wright, J. F., Nathwani, A. C., Nienhuis, A. W., Davidoff, A. M., & Gray, J. T. (2012) · Human Gene Therapy Methods · doi:10.1089/hgtb.2011.104 · Cited by 97 · see citing works

Scalable Recombinant Adeno-Associated Virus Production Using Recombinant Herpes Simplex Virus Type 1 Coinfection of Suspension-Adapted Mammalian Cells

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Removal of empty capsids from type 1 adeno-associated virus vector stocks by anion-exchange chromatography potentiates transgene expression

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Scalable serum-free production of recombinant adeno-associated virus type 2 by transfection of 293 suspension cells

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A helper virus-free packaging system for recombinant adeno-associated virus vectors

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A Single-Step Affinity Column for Purification of Serotype-5 Based Adeno-associated Viral Vectors

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Endotoxin Detection – from Limulus Amebocyte Lysate to Recombinant Factor C

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AAV Empty Capsids: For Better or for Worse?

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Undetectable transcription of cap in a clinical AAV vector: implications for preformed capsid in immune responses

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Rep-Mediated Nicking of the Adeno-Associated Virus Origin Requires Two Biochemical Activities, DNA Helicase Activity and Transesterification

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A Versatile and Scalable Two-Step Ion-Exchange Chromatography Process for the Purification of Recombinant Adeno-associated Virus Serotypes-2 and -5

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Advanced characterization of DNA molecules in rAAV vector preparations by single-stranded virus next-generation sequencing

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Production of Recombinant Adeno-Associated Viral Vectors Using a Baculovirus/Insect Cell Suspension Culture System: From Shake Flasks to a 20-L Bioreactor

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Evidence for pH-Dependent Protease Activity in the Adeno-Associated Virus Capsid

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