Manufacturing of recombinant adeno-associated viruses using mammalian expression platforms

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

(2016) Elizabeth E. Pierson et al.   ANALYTICAL CHEMISTRY

Viral vectors for gene therapy and gene modification approaches

(2016) Otto-Wilhelm Merten et al.   BIOCHEMICAL ENGINEERING JOURNAL

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

(2016) Joshua C Grieger et al.   MOLECULAR THERAPY

Development and Optimization of AAV hFIX Particles by Transient Transfection in an iCELLis®Fixed-Bed Bioreactor

(2016) Alicia D. Powers et al.   Human Gene Therapy Methods

Manufacturing of recombinant adeno-associated viral vectors: new technologies are welcome

(2016) Eduard Ayuso   Molecular Therapy-Methods & Clinical Development

Manufacturing of recombinant adeno-associated viral vectors for clinical trials

(2016) Nathalie Clément et al.   Molecular Therapy-Methods & Clinical Development

Strategies to generate high-titer, high-potency recombinant AAV3 serotype vectors

(2016) Chen Ling et al.   Molecular Therapy-Methods & Clinical Development

A scalable method for the production of high-titer and high-quality adeno-associated type 9 vectors using the HSV platform

(2016) Laura Adamson-Small et al.   Molecular Therapy-Methods & Clinical Development

Rational plasmid design and bioprocess optimization to enhance recombinant adeno-associated virus (AAV) productivity in mammalian cells

(2015) Verena V. Emmerling et al.   Biotechnology Journal

Scalable Downstream Strategies for Purification of Recombinant Adeno- Associated Virus Vectors in Light of the Properties

(2015) Weihong Qu et al.   CURRENT PHARMACEUTICAL BIOTECHNOLOGY

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

(2015) Brenda Burnham et al.   Human Gene Therapy Methods

Stability and Compatibility of Recombinant Adeno-Associated Virus Under Conditions Commonly Encountered in Human Gene Therapy Trials

(2015) Alisha M. Gruntman et al.   Human Gene Therapy Methods

Development of a Rapid, Robust, and Universal PicoGreen-Based Method to Titer Adeno-Associated Vectors

(2015) Jose Piedra et al.   Human Gene Therapy Methods

Rational plasmid design and bioprocess optimization to enhance recombinant adeno-associated virus (AAV) productivity in mammalian cells

(2015) Verena V. Emmerling et al.   Biotechnology Journal

Advanced Characterization of DNA Molecules in rAAV Vector Preparations by Single-stranded Virus Next-generation Sequencing

(2015) Emilie Lecomte et al.   Molecular Therapy-Nucleic Acids

Simple downstream process based on detergent treatment improves yield and in vivo transduction efficacy of adeno-associated virus vectors

(2015) Gabriella Dias Florencio et al.   Molecular Therapy-Methods & Clinical Development

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

(2014) Balaji Balakrishnan et al.   CURRENT GENE THERAPY

AAV Empty Capsids: For Better or for Worse?

(2014) J Fraser Wright   MOLECULAR THERAPY

Proteomics Analysis of Co-Purifying Cellular Proteins Associated with rAAV Vectors

(2014) Biao Dong et al.   PLoS One

Immune Responses to AAV-Vectors, the Glybera Example from Bench to Bedside

(2014) Valerie Ferreira et al.   Frontiers in Immunology

A simplified purification protocol for recombinant adeno-associated virus vectors

(2014) Mark Potter et al.   Molecular Therapy-Methods & Clinical Development

Immune responses to AAV vectors: overcoming barriers to successful gene therapy

(2013) F. Mingozzi et al.   BLOOD

A novel and rapid method to determine doxycycline in human plasma by liquid chromatography tandem mass spectrometry

(2013) AChaitanya Krishna et al.   INDIAN JOURNAL OF PHARMACEUTICAL SCIENCES

CpG-depleted adeno-associated virus vectors evade immune detection

(2013) Susan M. Faust et al.   JOURNAL OF CLINICAL INVESTIGATION

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

(2013) Parminder Singh Chahal et al.   JOURNAL OF VIROLOGICAL METHODS

Comparative Analysis of Adeno-Associated Virus Capsid Stability and Dynamics

(2013) V. Rayaprolu et al.   JOURNAL OF VIROLOGY

Generation and Characterization of Adeno-Associated Virus Producer Cell Lines for Research and Preclinical Vector Production

(2013) John Martin et al.   Human Gene Therapy Methods

AAV2/8 Vectors Purified from Culture Medium with a Simple and Rapid Protocol Transduce Murine Liver, Muscle, and Retina Efficiently

(2013) Monica Doria et al.   Human Gene Therapy Methods

Absolute Determination of Single-Stranded and Self-Complementary Adeno-Associated Viral Vector Genome Titers by Droplet Digital PCR

(2013) Martin Lock et al.   Human Gene Therapy Methods

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

(2012) E. D. Horowitz et al.   JOURNAL OF VIROLOGY

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

(2012) M. Salganik et al.   JOURNAL OF VIROLOGY

Successful Regional Delivery and Long-term Expression of a Dystrophin Gene in Canine Muscular Dystrophy: A Preclinical Model for Human Therapies

(2012) Zejing Wang et al.   MOLECULAR THERAPY

Analysis of Particle Content of Recombinant Adeno-Associated Virus Serotype 8 Vectors by Ion-Exchange Chromatography

(2012) Martin Lock et al.   Human Gene Therapy Methods

Systemic Errors in Quantitative Polymerase Chain Reaction Titration of Self-Complementary Adeno-Associated Viral Vectors and Improved Alternative Methods

(2012) Paolo Fagone et al.   Human Gene Therapy Methods

Quantification of AAV Particle Titers by Infrared Fluorescence Scanning of Coomassie-Stained Sodium Dodecyl Sulfate–Polyacrylamide Gels

(2012) Erik Kohlbrenner et al.   Human Gene Therapy Methods

Evaluation of a Droplet Digital Polymerase Chain Reaction Format for DNA Copy Number Quantification

(2011) Leonardo B. Pinheiro et al.   ANALYTICAL CHEMISTRY

Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations

(2011) Samuel G. Jacobson   ARCHIVES OF OPHTHALMOLOGY

Systemic Elimination of de novo Capsid Protein Synthesis from Replication-Competent AAV Contamination in the Liver

(2011) Hui Lu et al.   HUMAN GENE THERAPY

Good Manufacturing Practice Production of Self-Complementary Serotype 8 Adeno-Associated Viral Vector for a Hemophilia B Clinical Trial

(2011) James A. Allay et al.   HUMAN GENE THERAPY

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

(2011) Sylvain Cecchini et al.   HUMAN GENE THERAPY

Phase 2 Clinical Trial of a Recombinant Adeno-Associated Viral Vector Expressing α1-Antitrypsin: Interim Results

(2011) Terence R. Flotte et al.   HUMAN GENE THERAPY

Mycoplasma testing of cell substrates and biologics: Review of alternative non-microbiological techniques

(2011) Dmitriy V. Volokhov et al.   MOLECULAR AND CELLULAR PROBES

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

(2011) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Clearance and characterization of residual HSV DNA in recombinant adeno-associated virus produced by an HSV complementation system

(2010) G-J Ye et al.   GENE THERAPY

Characterization of a Recombinant Adeno-Associated Virus Type 2 Reference Standard Material

(2010) Martin Lock et al.   HUMAN GENE THERAPY

Efficient Serotype-Dependent Release of Functional Vector into the Culture Medium During Adeno-Associated Virus Manufacturing

(2010) Luk H. Vandenberghe et al.   HUMAN GENE THERAPY

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

(2010) Martin Lock et al.   HUMAN GENE THERAPY

A Versatile Adeno-Associated Virus Vector Producer Cell Line Method for Scalable Vector Production of Different Serotypes

(2010) Zhenhua Yuan et al.   HUMAN GENE THERAPY

A pseudo-plaque method for infectious particle assay and clonal isolation of adeno-associated virus

(2010) Daniel A.J. Mitchell et al.   JOURNAL OF VIROLOGICAL METHODS

Induction of immune tolerance to FIX by intramuscular AAV gene transfer is independent of the activation status of dendritic cells

(2009) A. S. Bharadwaj et al.   BLOOD

High AAV vector purity results in serotype- and tissue-independent enhancement of transduction efficiency

(2009) E Ayuso et al.   GENE THERAPY

Manufacturing Recombinant Adeno-Associated Viral Vectors from Producer Cell Clones

(2009) Barbara A. Thorne et al.   HUMAN GENE THERAPY

A Sensitive Cell-Based Assay to Measure the Doxycycline Concentration in Biological Samples

(2009) Wendy Kleibeuker et al.   HUMAN GENE THERAPY

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

(2009) Takashi Okada et al.   HUMAN GENE THERAPY

Transient Transfection Methods for Clinical Adeno-Associated Viral Vector Production

(2009) J. Fraser Wright   HUMAN GENE THERAPY

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

(2009) Darby L. Thomas et al.   HUMAN GENE THERAPY

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

(2009) Nathalie Clément et al.   HUMAN GENE THERAPY

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

(2009) Richard H Smith et al.   MOLECULAR THERAPY

Characterization of Genome Integrity for Oversized Recombinant AAV Vector

(2009) Biao Dong et al.   MOLECULAR THERAPY

Effect of Genome Size on AAV Vector Packaging

(2009) Zhijian Wu et al.   MOLECULAR THERAPY

An efficient rHSV-based complementation system for the production of multiple rAAV vector serotypes

(2008) W Kang et al.   GENE THERAPY

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

(2008) J F Wright   GENE THERAPY

Biodistribution and Safety Profile of Recombinant Adeno-Associated Virus Serotype 6 Vectors following Intravenous Delivery

(2008) D. Stone et al.   JOURNAL OF VIROLOGY

Self-complementary AAV Vectors; Advances and Applications

(2008) Douglas M McCarty   MOLECULAR THERAPY

Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection

(2008) Carmela Zincarelli et al.   MOLECULAR THERAPY

Undetectable Transcription of cap in a Clinical AAV Vector: Implications for Preformed Capsid in Immune Responses

(2008) Bernd Hauck et al.   MOLECULAR THERAPY

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

(2005) Jamal Meghrous et al.   BIOTECHNOLOGY PROGRESS