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

Allay, James A., Sleep, Susan, Long, Scott, Tillman, David M., Clark, Rob, Carney, Gael, Fagone, Paolo, McIntosh, Jenny H. +4 more·2011·Human Gene Therapy
DOI 10.1089/hum.2010.202

Cited by (152)

Items 101–152 of 152

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  8. Safety and efficacy evaluations of an adeno-associated virus variant for preparing IL10-secreting human neural stem cell-based therapeutics
    Mira Cho, Kwangsoo Jung, Seung‐Hyun Kim, Il‐Sun Kim, Miri Kim, Mikyung Shin, Haeshin Lee, Kook In Park, Jae‐Hyung Jang · 2019 · Gene Therapy
  9. Approaches to purification and concentration of rAAV vectors for gene therapy
    Lyubava Belova, Konstantin S. Kochergin-Nikitsky, Anastasia Erofeeva, А. В. Лавров, Svetlana Smirnikhina · 2022 · BioEssays
  10. Utilizing adeno‐associated virus as a vector in treating genetic disorders or human cancers
    Fu‐Hsuan Shih, Hsiung-Hao Chang, Yi‐Ching Wang · 2024 · IUBMB Life
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    Paul Edward Monahan, Tong Gui · 2013 · Current Opinion in Hematology
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    Yuxia Luo, Amy Frederick, John Munson Martin, Abraham Scaria, Seng H. Cheng, Donna Armentano, Samuel C. Wadsworth, Karen A. Vincent · 2017 · Human Gene Therapy Methods
  13. Interspecies normalization of dose‐response relationship for adeno‐associated virus‐mediated haemophilia gene therapy—Application to human dose prediction
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  14. Analysis of the impact of pluronic acid on the thermal stability and infectivity of AAV6.2FF
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  15. Potential limits of AAV‐based gene therapy with the use of new transgenes expressing factor IX fusion proteins
    Sandra Le Quellec, Allison P. Dane, Nathalie Enjolras, Jenny McIntosh, Cecilia Rosales, Claude Négrier, Amit Chunilal Nathwani · 2018 · Haemophilia
  16. Enhancement of Recombinant Adeno‐Associated Virus Production in HEK293 Cells by Application of Small Molecule Additives and Genetic Regulation Based on Perfusion Technique
    Yang Sun, Tingqi Yang, Xiaotong Liu, Jianqi Nie, Kuiqi Jin, Hua Li, Pei Zhou, Yinbiao Xu, Yupeng Liu, Zhonghu Bai · 2025 · Biotechnology Journal
  17. Adeno-associated virus vector-based gene therapy for hereditary diseases: current problems of application and approaches to solve them
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  18. Rapid and Noninvasive Quantification of Capsid Gene Filling Level Using Water Proton Nuclear Magnetic Resonance
    Marc B. Taraban, Michael T. Jones, Yihua Bruce Yu · 2021 · Analytical Chemistry
  19. Spectral Interferences Impede the High-Resolution Mass Analysis of Recombinant Adeno-Associated Viruses
    Victor C. Yin, Paul W. A. Devine, Janet C. Saunders, Alistair R. Hines, Sam Shepherd, Marcin Dembek, Claire Dobson, Joost Snijder, Nicholas J. Bond, Albert J. R. Heck · 2022 · bioRxiv (Cold Spring Harbor Laboratory)
  20. Trends in orally viral vector gene delivery and therapy
    Jingqi Xie, Ruian Xu · 2017 · Elsevier eBooks
  21. Systemic Errors in Quantitative Polymerase Chain Reaction Titration of Self-Complementary Adeno-Associated Viral Vectors and Improved Alternative Methods
    Paolo Fagone, John Fraser Wright, Amit Chunilal Nathwani, Arthur W. Nienhuis, Andrew M. Davidoff, John T. Gray · 2011 · Human Gene Therapy
  22. Implementation of the quality-by-design concept for an adeno-associated viral vector-based gene therapy
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  23. An SDS-NaOH-based method to isolate genome of recombinant adeno-associated virus vectors for physical titer measurement
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  30. Optimizing LOD/LOQ and sample consumption during analytical ultracentrifugation to characterize AAV gene therapy vectors
    Klaus Richter, Christine Wurm, Andrea Hawe, Tim Menzen, Kim Strasser, Jana Bauer, Michael Salomon, Shawn M. Sternisha, Ross VerHeul, Akash Bhattacharya · 2026 · Journal of Pharmaceutical and Biomedical Analysis
  31. The Use of Viral Vectors to Enhance Cognition
    Tsuneya Ikezu · 2015 · Elsevier eBooks
  32. Gene Therapy in Cartilage Repair and Regeneration
    Sheri Wang, Bing Wang · 2018 · Springer eBooks
  33. Adeno-Associated Virus Vectors for Gene Therapy: From Scalable Production Systems via Serotype-Specific Glycan Interaction Patterns to Gene Transfer Applications
    Mario Mietzsch · 2014
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  35. A Translational Pathway for Recombinant Adeno-Associated Virus Human Gene Therapy: From Target Identification and Animal Modeling of the Disease to Non-Human Primate and Human Studies
    Alisha M. Gruntman · 2016 · The Journal of the American Medical Association (JAMA) Network (American Medical Association)
  36. Empty Capsids in Production of Viral Vector: Separation and Reassembly
    Xinghua Peng, Nazmul Karim M · 2018 · Journal of Bioengineering and Biomedical Sciences
  37. Minimally invasive routes of AAV administration to treat GM1 gangliosidosis
    Amanda L. Gross · 2020
  38. Faculty Opinions recommendation of Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.
    Lucio Luzzatto · 2012 · Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature
  39. Strategies for Hemophilia Treatment, a literature review of current evidence
    Fahimeh Ghasemi, Nafiseh Erfanian, Samira Karbasi, Alireza Zomorodipour · 2023 · Sālnāmah va āmār.
  40. Non-encapsidated miRNA contaminants found in AAV preparations
    Mark A. Brimble, Stephen M. Winston · 2024 · Molecular Therapy — Methods & Clinical Development
  41. CMC Considerations for Continuous Bioprocess Design, Development, and Manufacturing
    Yanhuai Richard Ding, Margaret Peggy Marino · 2024 · Springer eBooks
  42. 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
  43. Additional file 1 of Analysis of the impact of pluronic acid on the thermal stability and infectivity of AAV6.2FF
    Sylvia P. Thomas, Marcus M. Spinelli, Amira D. Rghei, Jordyn A. Lopes, Nicole Zielinska, Benjamin M. McLeod, Yanlong Pei, Zhang, Wei, Bernard Thébaud, Khalil Karimi et al. · 2024 · Figshare
  44. Rational engineering of the P5 TRS-mimic site and REP78/68 start codon yields promoter variants that improve rAAV purity while maintaining high titers
    Stephen M. Winston, Mark A. Brimble, Kristin Wiggins, Isaiah L. Reeves, Yunyu Spence, Baochang Fan, Shaoyuan Tan, Christopher L. Morton, Pei-Hsin Cheng, Mary Y. Spence et al. · 2026 · Molecular Therapy Advances
  45. Characterisation of AAV capsid-specific CD8+ T cell responses in human subjects
    Céline Vandamme · 2016 · theses.fr (ABES)
  46. Characterization of rAAV vectors packaging in baculovirusinfected insect cells
    Magalie Penaud‐Budloo · 2018 · theses.fr (ABES)
  47. When engineering new recombinant factor IX molecules meets gene therapy : improvement of factor IX plasma level in patients with haemophilia B?
    Sandra Le Quellec · 2018 · theses.fr (ABES)
  48. 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)
  49. 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
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  51. Comparison of Wildtype and Recombinant Adeno‐Associated Virus (AAV) Production Reveals Distinct Productivity and Content Ratio Profiles
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  52. Vectors for therapeutic antisense sequences delivery and the modification of messenger RNA processing
    Julie Tordo · 2013 · UCL Discovery (University College London)