Analytical methods for process and product characterization of recombinant adeno-associated virus-based gene therapies
出版年份 2021 全文链接
标题
Analytical methods for process and product characterization of recombinant adeno-associated virus-based gene therapies
作者
关键词
gene therapies, viral vectors, adeno-associated virus, AAV, critical quality attributes, CQAs, process characterization, process understanding, process development
出版物
Molecular Therapy-Methods & Clinical Development
Volume 20, Issue -, Pages 740-754
出版商
Elsevier BV
发表日期
2021-02-18
DOI
10.1016/j.omtm.2021.02.010
参考文献
相关参考文献
注意：仅列出部分参考文献，下载原文获取全部文献信息。- Engineering adeno-associated virus vectors for gene therapy
- (2020) Chengwen Li et al. NATURE REVIEWS GENETICS
- Quantification of Adeno-Associated Virus with Safe Nucleic Acid Dyes
- (2020) Jian Xu et al. HUMAN GENE THERAPY
- Holographic Imaging of Insect Cell Cultures: Online Non-Invasive Monitoring of Adeno-Associated Virus Production and Cell Concentration
- (2020) Daniel A. M. Pais et al. Processes
- Optimization of the quality by design approach for gene therapy products: a case study for adeno-associated viral vectors
- (2020) Toshimitsu Tanaka et al. EUROPEAN JOURNAL OF PHARMACEUTICS AND BIOPHARMACEUTICS
- Single molecule mass photometry of nucleic acids
- (2020) Yiwen Li et al. NUCLEIC ACIDS RESEARCH
- Creation of a High-Yield AAV Vector Production Platform in Suspension Cells Using a Design-of-Experiment Approach
- (2020) Huiren Zhao et al. Molecular Therapy-Methods & Clinical Development
- Clinical Development of Gene Therapies: The First Three Decades and Counting.
- (2020) Larissa Lapteva et al. Molecular Therapy-Methods & Clinical Development
- Stability of the adeno-associated virus 8 reference standard material
- (2019) Magalie Penaud-Budloo et al. GENE THERAPY
- 2D droplet digital PCR as a tool for titration and integrity evaluation of recombinant adeno-associated viral vectors
- (2019) Birei Furuta-Hanawa et al. Human Gene Therapy Methods
- Virus-like particle size and molecular weight/mass determination applying gas-phase electrophoresis (native nES GEMMA)
- (2019) Victor U. Weiss et al. ANALYTICAL AND BIOANALYTICAL CHEMISTRY
- Enabling PAT in insect cell bioprocesses: In situ monitoring of recombinant adeno‐associated virus production by fluorescence spectroscopy
- (2019) Daniel A. M. Pais et al. BIOTECHNOLOGY AND BIOENGINEERING
- Accurate Quantification and Characterization of Adeno-Associated Viral Vectors
- (2019) David Dobnik et al. Frontiers in Microbiology
- Achieving High-Yield Production of Functional AAV5 Gene Delivery Vectors via Fedbatch in an Insect Cell-One Baculovirus System
- (2019) Pranav R.H. Joshi et al. Molecular Therapy-Methods & Clinical Development
- Filling AAV capsids: estimating success by cryo-EM
- (2019) Suriyasri Subramanian et al. HUMAN GENE THERAPY
- Analytical Strategies for Quantification of Adeno-Associated Virus Empty Capsids to Support Process Development
- (2019) Xiaotong Fu et al. Human Gene Therapy Methods
- Developing an Anion Exchange Chromatography (AEX) Assay for Determining Empty and Full Capsid Contents in AAV6.2
- (2019) Chunlei Wang et al. Molecular Therapy-Methods & Clinical Development
- Production of adeno-associated virus vectors for in vitro and in vivo applications
- (2019) Toyokazu Kimura et al. Scientific Reports
- The approved gene therapy drugs worldwide: from 1998 to 2019
- (2019) Cui-Cui Ma et al. BIOTECHNOLOGY ADVANCES
- Adeno-associated virus capsid protein expression in Escherichia coli and chemically defined capsid assembly
- (2019) Dinh To Le et al. Scientific Reports
- Flow virometry as a tool to study viruses
- (2018) J. Lizbeth Reyes Zamora et al. METHODS
- Quantitative mass imaging of single biological macromolecules
- (2018) Gavin Young et al. SCIENCE
- Pharmacology of Recombinant Adeno-associated Virus Production
- (2018) Magalie Penaud-Budloo et al. Molecular Therapy-Methods & Clinical Development
- Universal Method for the Purification of Recombinant AAV Vectors of Differing Serotypes
- (2018) Shelley A. Nass et al. Molecular Therapy-Methods & Clinical Development
- Adeno-associated Virus Genome Population Sequencing Achieves Full Vector Genome Resolution and Reveals Human-Vector Chimeras
- (2018) Phillip W.L. Tai et al. Molecular Therapy-Methods & Clinical Development
- Accurate Titration of Infectious AAV Particles Requires Measurement of Biologically Active Vector Genomes and Suitable Controls
- (2018) Achille François et al. Molecular Therapy-Methods & Clinical Development
- Highly Efficient Ultracentrifugation-free Chromatographic Purification of Recombinant AAV Serotype 9
- (2018) Taro Tomono et al. Molecular Therapy-Methods & Clinical Development
- Scalable Production of AAV Vectors in Orbitally Shaken HEK293 Cells
- (2018) Daniel Blessing et al. Molecular Therapy-Methods & Clinical Development
- Manufacturing Clinical Grade Recombinant Adeno-Associated Virus Using Invertebrate Cell Lines
- (2017) Robert M. Kotin et al. HUMAN GENE THERAPY
- Direct Head-to-Head Evaluation of Recombinant Adeno-associated Viral Vectors Manufactured in Human versus Insect Cells
- (2017) Oleksandr Kondratov et al. MOLECULAR THERAPY
- Protocol for Efficient Generation and Characterization of Adeno-Associated Viral Vectors
- (2017) Andreas Jungmann et al. Human Gene Therapy Methods
- Improving the Quality of Adeno-Associated Viral Vector Preparations: The Challenge of Product-Related Impurities
- (2017) Maria Schnödt et al. Human Gene Therapy Methods
- Accurate Identification and Quantification of DNA Species by Next-Generation Sequencing in Adeno-Associated Viral Vectors Produced in Insect Cells
- (2017) Magalie Penaud-Budloo et al. Human Gene Therapy Methods
- Universal label-free in-process quantification of influenza virus-like particles
- (2017) Sofia B. Carvalho et al. Biotechnology Journal
- Manufacturing of recombinant adeno-associated viruses using mammalian expression platforms
- (2017) Marc-André Robert et al. Biotechnology Journal
- Label-Free Single-Molecule Imaging with Numerical-Aperture-Shaped Interferometric Scattering Microscopy
- (2017) Daniel Cole et al. ACS Photonics
- Droplet Digital PCR versus qPCR for gene expression analysis with low abundant targets: from variable nonsense to publication quality data
- (2017) Sean C. Taylor et al. Scientific Reports
- Comparison of biosensor platforms in the evaluation of high affinity antibody-antigen binding kinetics
- (2016) Danlin Yang et al. ANALYTICAL BIOCHEMISTRY
- Resolving Adeno-Associated Viral Particle Diversity With Charge Detection Mass Spectrometry
- (2016) Elizabeth E. Pierson et al. ANALYTICAL CHEMISTRY
- Determination of critical quality attributes for monoclonal antibodies using quality by design principles
- (2016) Nadja Alt et al. BIOLOGICALS
- High-Density Recombinant Adeno-Associated Viral Particles are Competent Vectors for In Vivo Transduction
- (2016) Qizhao Wang et al. HUMAN GENE THERAPY
- 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
- Manufacturing of recombinant adeno-associated viral vectors for clinical trials
- (2016) Nathalie Clément et al. Molecular Therapy-Methods & Clinical Development
- Rapid quantitation of porcine epidemic diarrhea virus (PEDV) by Virus Counter
- (2015) Xiangbin Zhang et al. JOURNAL OF VIROLOGICAL METHODS
- Evaluation of ViroCyt® Virus Counter for Rapid Filovirus Quantitation
- (2015) Cynthia Rossi et al. Viruses-Basel
- Systematic Comparison and Validation of Quantitative Real-Time PCR Methods for the Quantitation of Adeno-Associated Viral Products
- (2015) Natalie Jayne Werling et al. Human Gene Therapy Methods
- Analytical Ultracentrifugation as an Approach to Characterize Recombinant Adeno-Associated Viral Vectors
- (2015) Brenda Burnham et al. Human Gene Therapy Methods
- FDA Statement Regarding the Use of Adeno-Associated Virus Reference Standard Materials
- (2015) Denise K. Gavin 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
- Understanding Pharmaceutical Quality by Design
- (2014) Lawrence X. Yu et al. AAPS Journal
- Manufacturing and Characterization of a Recombinant Adeno-Associated Virus Type 8 Reference Standard Material
- (2014) Eduard Ayuso et al. HUMAN GENE THERAPY
- Weighing nanoparticles in solution at the attogram scale
- (2014) S. Olcum et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Therapeutic protein aggregation: mechanisms, design, and control
- (2014) Christopher J. Roberts TRENDS IN BIOTECHNOLOGY
- 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
- Characterization and Quantitation of Aggregates and Particles in Interferon-β Products: Potential Links Between Product Quality Attributes and Immunogenicity
- (2012) James G. Barnard et al. JOURNAL OF PHARMACEUTICAL SCIENCES
- Biophysical and Ultrastructural Characterization of Adeno-Associated Virus Capsid Uncoating and Genome Release
- (2012) E. D. Horowitz et al. JOURNAL OF VIROLOGY
- 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
- Limitations of Encapsidation of Recombinant Self-Complementary Adeno-Associated Viral Genomes in Different Serotype Capsids and Their Quantitation
- (2012) Yuan Wang 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
- High-Throughput Droplet Digital PCR System for Absolute Quantitation of DNA Copy Number
- (2011) Benjamin J. Hindson et al. ANALYTICAL CHEMISTRY
- 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
- Evaluation of electrospray differential mobility analysis for virus particle analysis: Potential applications for biomanufacturing
- (2011) Suvajyoti Guha et al. JOURNAL OF VIROLOGICAL METHODS
- Physical analysis of virus particles using electrospray differential mobility analysis
- (2011) Leonard F. Pease TRENDS IN BIOTECHNOLOGY
- Production, Purification and Characterization of Adeno-Associated Vectors
- (2010) Eduard Ayuso et al. CURRENT GENE THERAPY
- Characterization of a Recombinant Adeno-Associated Virus Type 2 Reference Standard Material
- (2010) Martin Lock et al. HUMAN GENE THERAPY
- Strategies for the Assessment of Protein Aggregates in Pharmaceutical Biotech Product Development
- (2010) John den Engelsman et al. PHARMACEUTICAL RESEARCH
- Precision of protein aggregation measurements by sedimentation velocity analytical ultracentrifugation in biopharmaceutical applications
- (2009) John P. Gabrielson et al. ANALYTICAL BIOCHEMISTRY
- A Critical Review of Methods for Size Characterization of Non-Particulate Protein Aggregates
- (2009) John Philo CURRENT PHARMACEUTICAL BIOTECHNOLOGY
- Potential inaccurate quantitation and sizing of protein aggregates by size exclusion chromatography: Essential need to use orthogonal methods to assure the quality of therapeutic protein products
- (2009) John F. Carpenter et al. JOURNAL OF PHARMACEUTICAL SCIENCES
- Bioprocess optimization using design-of-experiments methodology
- (2008) Carl-Fredrik Mandenius et al. BIOTECHNOLOGY PROGRESS
- Manufacturing and characterizing AAV-based vectors for use in clinical studies
- (2008) J F Wright GENE THERAPY
- Undetectable Transcription of cap in a Clinical AAV Vector: Implications for Preformed Capsid in Immune Responses
- (2008) Bernd Hauck et al. MOLECULAR THERAPY
Discover Peeref hubs
Discuss science. Find collaborators. Network.
Join a conversationCreate your own webinar
Interested in hosting your own webinar? Check the schedule and propose your idea to the Peeref Content Team.
Create Now