Pharmacology of Recombinant Adeno-associated Virus Production

Penaud-Budloo, Magalie, François, Achille, Clément, Nathalie, Ayuso, Eduard·2018·Molecular Therapy - Methods & Clinical Development

Cited by (177)

Items 101–177 of 177

  1. Strategies and mechanisms of neuronal reprogramming
    Yue Wan, Yan Ping Ding · 2023 · Brain Research Bulletin
  2. Assessment of Residual Full-Length SV40 Large T Antigen in Clinical-Grade Adeno-Associated Virus Vectors Produced in 293T Cells
    Bishnu P. De, Sara Cram, Hyunmi Lee, Jonathan B. Rosenberg, Dolan Sondhi, Ronald G. Crystal, Stephen M. Kaminsky · 2023 · Human Gene Therapy
  3. Recombinant adeno‐associated virus production evaluation in Chinese hamster ovary cells
    Thu Cao, Dayue Chen, Gavin C. Barnard, Amy Y. Shen · 2023 · Biotechnology and Bioengineering
  4. A simple and sensitive differential digestion method to analyze adeno-associated virus residual host cell proteins by LC-MS
    Yunli Hu, Mengqi Hu, Xiang Ye, Zhijie Wu, Jianming Kang, Christina Wong, Nisha T. Palackal, Haibo Qiu, Ning Li · 2024 · Journal of Pharmaceutical and Biomedical Analysis
  5. Protocol for producing an adeno-associated virus vector by controlling capsid expression timing
    Kenji Ohba, Hiroaki Mizukami · 2023 · STAR Protocols
  6. Manufacturing and Quality of Advanced Therapeutics: Cellular Therapies, CAR-T and Gene Therapies
    Christian van den Bos, Robert Keefe, Mary Keville, Michael T. McCaman · 2023 · Cell engineering
  7. Inhalable and Nasal Biologics: Analytical, Formulation, Development, and Regulatory Considerations
    David C. Cipolla, Christopher J. Gruenloh, Nani P. Kadrichu, Philip J. Kuehl, Lei Mao, Bing Li, Svetlana Lyapustina, Heidi M. Mansour, Franz‐Josef K. Rehmann, Irene Rossi et al. · 2025 · Journal of Aerosol Medicine and Pulmonary Drug Delivery
  8. Safety and Tolerability of the Adeno-Associated Virus Vector, AAV6.2FF, Expressing a Monoclonal Antibody in Murine and Ovine Animal Models
    Amira D. Rghei, Laura P. van Lieshout, Benjamin M. McLeod, Yanlong Pei, Jordyn A. Lopes, Nicole Zielinska, Enzo M. Baracuhy, Brenna A. Y. Stevens, Sylvia P. Thomas, Jacob G. E. Yates et al. · 2021 · Biomedicines
  9. Impact of dual‐baculovirus infection on the Sf9 insect cell transcriptome during rAAV production using single‐cell RNA‐seq
    Nikolaus Virgolini, Marco Silvano, Ryan Hagan, Ricardo Correia, Paula M. Alves, Colin Clarke, António Roldão, Inês A. Isidro · 2023 · Biotechnology and Bioengineering
  10. Querying Recombination Junctions of Replication-Competent Adeno-Associated Viruses in Gene Therapy Vector Preparations with Single Molecule, Real-Time Sequencing
    Mitchell Yip, Jing Chen, Yan Zhi, Ngoc Tam Tran, Suk Namkung, Eric Pastor, Guangping Gao, Phillip W.L. Tai · 2023 · Viruses
  11. Improved endotoxin removal using ecofriendly detergents for intensified plasmid capture
    André Kiesewetter, Akshat Gupta, Anja Heinen‐Kreuzig, Trish Greenhalgh, Andreas Stein · 2023 · Biotechnology Progress
  12. The Pharmacology of Gene and Cell Therapy
    Thomas J. Conlon, Fulvio Mavilio · 2018 · Molecular Therapy — Methods & Clinical Development
  13. Liver gene transfer for metabolite detoxification in inherited metabolic diseases
    Alfonso Manuel D’Alessio, Iolanda Boffa, Lucia De Stefano, Leandro R. Soria, Nicola Brunetti‐Pierri · 2024 · FEBS Letters
  14. Unraveling Cytotoxicity in HEK293 Cells During Recombinant AAV Production for Gene Therapy Applications
    Pranay Ladiwala, Nelson Ndahiro, Pricila Hauk, Junneng Wen, Justin Sargunas, Yu‐Ju Chen, Erik S. Barton, Michael J. Betenbaugh · 2025 · Biotechnology Journal
  15. Electrophoresis-Mediated Characterization of Full and Empty Adeno-Associated Virus Capsids
    Adriana Coll De Peña, Lucy Masto, James Atwood, Anubhav Tripathi · 2022 · ACS Omega
  16. Concise Analysis of Single-Stranded DNA of Recombinant Adeno-Associated Virus By Automated Electrophoresis System
    Yuzhe Yuan, Kiyoko Higashiyama, Noriko Hashiba, Kyoko Masumi‐Koizumi, Keisuke Yusa, Kazuhisa Uchida · 2023 · Human Gene Therapy
  17. Profiling AAV vector heterogeneity & contaminants using next-generation sequencing methods
    Ngoc Tam Tran, Phillip W.L. Tai · 2024 · Cell and Gene Therapy Insights
  18. Efficient adeno-associated virus serotype 5 capture with affinity functionalized nanofiber adsorbents
    Salomé Neto, João P. Mendes, Susana B. Dos Santos, Anita Solbrand, Manuel J.T. Carrondo, Cristina Peixoto, Ricardo J. S. Silva · 2023 · Frontiers in Bioengineering and Biotechnology
  19. Muscle overexpression of Klf15 via an AAV8-Spc5-12 construct does not provide benefits in spinal muscular atrophy mice
    N. Ahlskog, D. Hayler, A. Krueger, S. Kubinski, P. Claus, S. Hammond, M. J. Wood, R. Yáñez-Muñoz, M. Bowerman · 2019 · Gene Therapy
  20. Mammalian Stable Cell Platforms for Recombinant Adeno-associated Virus (rAAV) Production: Development Strategies and their Impact on Viral Productivity
    Sofia Fernandes, Joana Diogo, Ana Sofia Coroadinha · 2023 · Current Gene Therapy
  21. Diagnostic and therapeutic potential of circular RNA in brain tumors
    K. Katsushima, Kandarp Joshi, Ranjan J. Perera · 2023 · Neuro-Oncology Advances
  22. Characterization of residual microRNAs in AAV vector batches produced in HEK293 mammalian cells and Sf9 insect cells
    Magalie Penaud‐Budloo, Émilie Lecomte, Quentin Lecomte, Simon Pacouret, Frédéric Broucque, Aurélien Guy‐Duché, Jean-Baptiste Dupont, Laurence Jeanson, Cécile Robin, Véronique Blouin et al. · 2024 · Molecular Therapy — Methods & Clinical Development
  23. Characterization of Viral Genome Encapsidated in Adeno-associated Recombinant Vectors Produced in Yeast Saccharomyces cerevisiae
    Alvaro Galli, Ilenia Iaia, Maria Serena Milella, Filippo Cipriani, Veronica Della Latta, Mauro Giacca, Lorena Zentilin, Tiziana Cervelli · 2021 · Molecular Biotechnology
  24. Enrichment of Full AAV2 Using Multicolumn Countercurrent Solvent Gradient Purification (MCSGP)
    Julia M. Müller, Tereza Müllerová, Daniela Tobler, Damian Hauri, Ralf Plieninger, Yuki Higuchi, Ryosuke Takahashi, Sebastian Vogg, Thomas Müller‐Späth, Thomas K. Villiger · 2025 · Biotechnology and Bioengineering
  25. The whole‐genome sequencing of prevalent DENV‐1 strains during the largest dengue virus outbreak in Xishuangbanna Dai autonomous prefecture in 2019
    Daiying Li, Mingwang Long, Tingting Li, Yun Shu, Xiyun Shan, Juan Zhang, Dehong Ma, Shuying Long, Xiaodan Wang, Fan Jia et al. · 2022 · Journal of Medical Virology
  26. Production of VP3-only virus-like particles of Adeno-associated virus 2 in E. coli cells
    Chengyu Fu, Shruthi Gobbooru, Ashley T. Martino, Woon‐Kai Low · 2024 · Protein Expression and Purification
  27. Development of an AAV-RNAi strategy to silence the dominant variant GNAO1 c.607G>A linked to encephalopathy
    Evgenii A. Lunev, Natalia V. Klementieva, Svetlana G. Vassilieva, Egor A. Volovikov, David Jappy, Irina M. Savchenko, Ekaterina A. Svetlova, Anna V. Polikarpova, Maria Y. Shubina, Danil Spirin et al. · 2025 · Gene Therapy
  28. Deciphering Key Adenoviral Elements in the Production of Recombinant Adeno-Associated Virus Vectors
    Sofia Fernandes, Júlia Guerra, Mariana V. Ferreira, Ana Sofia Coroadinha · 2025 · Human Gene Therapy
  29. Adeno-associated virus vector-based gene therapy for hereditary diseases: current problems of application and approaches to solve them
    Tatiana V. Egorova, A. A. Piskunov, Dmitry A. Poteryaev · 2024 · Biological Products Prevention Diagnosis Treatment
  30. Considerations on Preclinical Neuromuscular Disease Gene Therapy Studies
    Dongsheng Duan · 2019 · Springer eBooks
  31. An ex vivo Approach to Assess Mitochondrial ROS by Flow Cytometry in AAV-tagged Astrocytes in Adult Mice
    Carlos Vicente‐Gutiérrez, Juan Pedro Bolaños · 2020 · BIO-PROTOCOL
  32. Reduced rAAV interference enhances rcAAV detection sensitivity
    Ming Yang, Sana Shaheen, Keying Yang, Xuxia Gao, Kaiwen Wu, Xiangying Zhu, Qian Kang, Lu Wang, Farhid Hemmatzadeh, Qingyun Zheng et al. · 2025 · Molecular Therapy — Methods & Clinical Development
  33. Suspension Culture Production and Purification of Adeno-Associated Virus by Iodixanol Density Gradient Centrifugation for In Vivo Applications
    Kathryn K. Harris, Oleksandr Kondratov, Sergei Zolotukhin · 2024 · Journal of Visualized Experiments
  34. Expression of Viral DNA Polymerase in Synthetic Recombinant Adeno‐Associated Virus Producer Cell Line Enhances Full Particle Productivity
    Yu‐Chieh Lin, Han‐Jung Kuo, Min Lu, Carissa Rungkittikhun, Wei‐Shou Hu · 2024 · Biotechnology and Bioengineering
  35. Adoptive T cell therapy: engineering and biomanufacturing chimeric antigen receptor-T cell
    Jackson P. Jenkins, Jaeyoung Park, Kristen Petersen, Kahmil Shajihan, Srivalli Kruthiventi, Michael J. Betenbaugh · 2018 · Current Opinion in Chemical Engineering
  36. Implementation of the quality-by-design concept for an adeno-associated viral vector-based gene therapy
    Копеин Дамир Сергеевич, G. N. Poroshin, Хамитов Равиль Авгатович · 2025 · Biological Products Prevention Diagnosis Treatment
  37. CRISPR-Cas9 engineering of CAR-T cells: Can non-viral nanoparticles unlock safer and scalable genome editing?
    Bruna My, Andrea Lia, Ludovica Rizzo, Gabriele Maiorano, Antonio Galeone, Ilaria Elena Palamà, Giuseppe Gigli · 2026 · iScience
  38. A clinician’s guide to AAV production – How manufacturing platforms shape vector properties
    Jonas Käsbach, Jørgen Barsett Magnus · 2025 · Medizinische Genetik
  39. Adeno-Associated Virus Vector Design–Moving the Adeno-Associated Virus to a Bioengineered Therapeutic Nanoparticle
    Nico Martin Jäschke, Hildegard Büning · 2022 · Hematology/Oncology Clinics of North America
  40. Molecular characterization of structural protein genes of dengue virus serotype 1 epidemic in Yunnan, Southwest China, in 2018
    Qingping Lan, Yun Shu, Linhao Li, Xiyun Shan, Dehong Ma, Tingting Li, Xiaodan Wang, Yue Pan, Junying Chen, Juan Zhang et al. · 2021 · Archives of Virology
  41. The CASwitch: a synthetic biology solution for high-performance inducible gene expression systems in biotechnology
    Giuliano De Carluccio, Virginia Fusco, Diego di Bernardo · 2023 · bioRxiv (Cold Spring Harbor Laboratory)
  42. Bio-Production of Adeno-Associated Virus for Gene Therapy
    Nicholas Donohue, Niamh Keogh, Stefano Boi, Niall Barron · 2021 · Cell engineering
  43. Chromatographic Purification of Viral Vectors for Gene Therapy Applications
    A Kearney · 2023 · Methods in molecular biology
  44. Particle size distribution resolution limit of AAV high-speed sedimentation velocity protocol relative to low-speed sedimentation velocity protocol, mass photometry, and charge-detection mass spectrometry
    Bashkim Kokona, Austin D. Vogt, Neal Whitaker, Aubree O’Neill, Kevin D. Ausman, Jin Wen, Alfredo Lopez De Leon, Kimberly A. Malecka, Thomas A. Laue, Steven A. Berkowitz et al. · 2025 · Journal of Pharmaceutical Sciences
  45. Significant Enhancement of Adeno‐Associated Virus Vector Yield for Gene Therapy Using Multidimensional Optimization Strategies
    Weiqiang Shen, Shuyuan Chen, Zhongwan Li, Wei Yu · 2025 · The FASEB Journal
  46. Removal of Empty AAV Capsids to Undetectable Levels Using Orthogonal Purification Steps
    Rahul D. Sheth, Sylvain Boutigny, Juan Perez, David Silberstein, Yanhong Zhang, Omair Karim, Geuncheol Gil, A K Rishi, Harmit Vora, Daniel Gold et al. · 2026 · Biotechnology and Bioengineering
  47. Stem Cell Based Exosomes: Are They Effective in Disease or Health?
    Meric Bilgic Kucukguven, Betül Çelebi‐Saltik · 2021 · Advances in experimental medicine and biology
  48. Ocular Gene Therapies
    Stylianos Michalakis, Maximilian-Joachim Gerhardt, Claudia Priglinger, Siegfried Georg Priglinger · 2021 · Springer eBooks
  49. Viral vectors for gene therapy
    Ang‐Chen Tsai, Silveli Suzuki‐Hatano, Christina A. Pacak · 2024 · Elsevier eBooks
  50. The End of the Beginning: The Journey to Molecular Therapies for Spinal Muscular Atrophy
    Christina A. Pacak, Peter B. Kang · 2019 · Pediatric Neurology
  51. Ocular Gene Therapies
    Stylianos Michalakis, Maximilian-Joachim Gerhardt, Claudia Priglinger, Siegfried Georg Priglinger · 2022 · Springer eBooks
  52. SSV-Seq 2.0, a more accurate PCR-free method for high-throughput sequencing of adeno-associated viral vector genomes
    Émilie Lecomte, Sylvie Saleün, Mathieu Bolteau, Aurélien Guy‐Duché, Oumeya Adjali, Véronique Blouin, Magalie Penaud‐Budloo, Eduard Ayuso · 2020
  53. Pricing Pressure of Advanced Therapy Medicinal Products can be Diminished by Contemporary Manufacturing Process Development
    Hanna P. Lesch · 2021 · Gene Technology
  54. Viral vector gene therapy approaches for regeneration and repair in spinal cord injury
    Simon J. O’Carroll, Connor A. Clemett · 2022 · Elsevier eBooks
  55. Characterization of Adeno-Associated Virus (AAV) Gene Therapy Products
    Anurag Singh Rathore, Jared R. Auclair, Anantdeep Kaur · 2023 · LCGC North America
  56. AAV-Vektoren – die imposante Karriere eines Parvovirus
    Nico Martin Jäschke, Hildegard Büning · 2023 · Springer eBooks
  57. Intrathecal Delivery of Viral Vector-Mediated Gene Therapy
    Orion P. Keifer · 2023 · Springer eBooks
  58. Conditional splicing system for tight control of viral overlapping genes
    Qing Yang, Jinlin Wang, Zhiwei Chen · 2024 · Journal of Virology
  59. Hope Unlocked: Gene Therapy with Viral Vectors and Gene Editing
    Jennifer J. Thiaville, Mariana Santana Dias, Sayuri E. M. Kato, Shashwati Basak, Srinivas Rengarajan, Hilda Petrs Silva, Natasha Rivas, Susan M. D’Costa · 2025 · Springer eBooks
  60. Regulatory Perspective for Biologics License Application of Recombinant Adeno-Associated Virus Products in China
    Jing Cui, Wenbo Wang, Dan Liu, Dongmei Han, Xiangwaner Jin, Wei Wei · 2025 · Human Gene Therapy
  61. The poly-(HA-GMA) hydrogel carrying AAV8-sTβRII alleviates scar formation in mice skin wound healing by inhibiting fibrosis
    Jinhao Chen, Lijun Zhan, Jinyan Duan, T Wang, Zenan Meng, Qianru Wang, Jianlin Yang, Xiaofei Huang, Yue Liao, Xinyu Song et al. · 2026 · Gene Therapy
  62. Compréhension de la neurophysiopathologie de l'ataxie de Friedreich et développement d'une approche de thérapie génique dans un nouveau modèle murin
    Charline de Montigny · 2018 · theses.fr (ABES)
  63. Thermostability of Adeno-Associated Virus (AAV) Vectors
    Simon Pacouret · 2018 · theses.fr (ABES)
  64. Etude de la réponse immunitaire virale préexistante anti-AAV chez l'homme
    Rebecca Xicluna · 2019 · theses.fr (ABES)
  65. Modification chimique d’AAV pour la thérapie génique
    Aurélien Leray · 2021 · theses.fr (ABES)
  66. Impact de l’inclusion du domaine C-terminal de la dystrophine sur l’efficacité d’une micro-dystrophine dans le cadre d’une thérapie génique de la Dystrophie Musculaire de Duchenne
    Audrey Bourdon · 2020 · theses.fr (ABES)
  67. Étude de stratégies thérapeutiques ciblant les anomalies calciques dans la Dystrophie Musculaire de Duchenne
    A. Creisméas · 2022 · theses.fr (ABES)
  68. Reducing encapsidated impurity DNA derived from plasmid backbone by modifying the p5 terminal resolution site in rAAV vector production
    Yuya Nishimura, Shoko Hataya, Shunsuke Saito, Naoki Makita · 2026 · bioRxiv (Cold Spring Harbor Laboratory)
  69. Manipulation of CA1 neuronal subtypes through Cre-mediated viral delivery in mice
    Dheeraj Songara, Hiyaa Singhee Ghosh · 2026 · bioRxiv (Cold Spring Harbor Laboratory)
  70. Insect-Cell-Produced AAV2 Vectors Activate Myeloid Innate Immune Response Featuring NETosis.
    Xiaomei Liu, Yi-Feng Zhou, Ziwei Tang, Wen-Jing Li, Shuang-Shi Wei, Kun Han · 2026 · Molecular Pharmaceutics
  71. YY1 Binding Motif Upstream of Rep/Cap Increases AAV Yield and Full Capsids
    Yuri Ofusa, Sanae Nishio, Tatsuji Enoki, Junichi Mineno, Keiya Ozawa, Hiroaki Mizukami, Kenji Ohba · 2026 · bioRxiv (Cold Spring Harbor Laboratory)
  72. AAV vector production in suspension cells using PEI transfection and sodium butyrate with orthogonal assessment of function and quality
    Sujan Ravendran, Simon Fammé, Maya Graham Noer, Thomas Wisbech Skov, Nanna S. Mikkelsen, Jessica Lee Schneller, Sofie Rahbek Dorset, Jonas Holst Wolff, Anaïs Marie Julie Møller, Didde Haslund et al. · 2026 · Molecular Therapy Advances
  73. MicroRNA-Based Therapeutic Strategies for Pulmonary Arterial Hypertension under Different Delivery Systems: Progress and Challenges
    Jiaxiang Chen, Xiaoyu Zhang, J N Zhang, Haobo Ren, Jiahui Xie, Runwei Ma · 2026 · Current Medicinal Chemistry
  74. Status and future of recombinant adeno‐associated virus vector manufacturing
    Frank Agbogbo, David J. Dismuke · 2026 · Biotechnology Progress
  75. Demonstration of nanofiltration‐based virus safety for AAV manufacturing using model viruses
    Brian Buesing, Tomoki Igi, Daniel Strauss · 2026 · Biotechnology Progress
  76. Development of a Novel, Eco‐Friendly Mechanical Cell Lysis Process Using Hollow Fiber Filter for AAV Manufacturing
    Mario Jabra, Ethan Butler, Junfen Ma, Bruno Figueroa, Yuanli Song · 2026 · Biotechnology and Bioengineering
  77. Advanced therapy medicinal products for local treatment using new delivery methods
    Marcin Zaradzki, Jasmin P. Soethoff, Stefanie Sauer, Anca Kliesow Remes, Oliver J. Müller, Andreas H. Wagner, Benedict Morath · 2026 · Biochemical Pharmacology