Long-term Safety and Efficacy Following Systemic Administration of a Self-complementary AAV Vector Encoding Human FIX Pseudotyped With Serotype 5 and 8 Capsid Proteins

Nathwani, Amit C, Rosales, Cecilia, McIntosh, Jenny, Rastegarlari, Ghasem, Nathwani, Devhrut, Raj, Deepak, Nawathe, Sushmita, Waddington, Simon N +16 more·2011·Molecular Therapy
DOI 10.1038/mt.2010.274

Cited by (345)

Items 301–345 of 345

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    Frank Jacobs, Stephanie C. Gordts, Bart De Geest · 2013 · Wiley eBooks
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  3. Progress and challenges in gene therapy for Crigler–Najjar syndrome
    Sem J. Aronson, Ulrich H. Beuers, Piter J. Bosma · 2015 · Expert Opinion on Orphan Drugs
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    Stuart A. Nicklin, Uta Griesenbach D, Andrew Howard Baker · 2015 · Human Gene Therapy
  5. Hemophilia B Gene Therapy in Humans Shows Promise
    Andrew D. Johnson · 2012 · Circulation Cardiovascular Genetics
  6. Emerging Gene Correction Strategies for Muscular Dystrophies: Scientific Progress and Regulatory Impact
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  7. Current strides in AAV-derived vectors and SIN channels further relieves the limitations of gene therapy
    Arome Solomon Odiba, Victoria Ottah, Oluchukwu Ogechukwu Anunobi, Chimere Young Ukegbu, Robert Ikechukwu Uroko, Comfort Ottah, Affiong Asuquo Edeke, Kingsley O. Omeje · 2017 · Egyptian Journal of Medical Human Genetics
  8. Promoting Neuroplasticity To Repair The Injured Spinal Cord
    Nina Weishaupt · 2013 · ERA: Education and Research Archive (University of Alberta)
  9. Systemic Delivery in Anti-aging Medicine: An Overview
    Yi Wang, Wing‐Fu Lai · 2020 · Healthy ageing and longevity
  10. Current Trends in the Gene Therapy of Hematologic Disorders
    Dragomira Nikolaeva Nikolova · 2021 · Acta Medica Bulgarica
  11. 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
  12. Gene Therapy
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  13. Faculty Opinions recommendation of Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction.
    Irina M. Conboy · 2018 · Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature
  14. Targeted local combination therapy with checkpoint inhibitors and CAR-NK cells in glioblastoma using DARPin-linked AAV vectors
    Maja Strecker · 2022
  15. Blood-brain barrier-directed gene therapy as a strategy to treat Niemann-Pick type C2 disease
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  16. Genome Integration of Adeno-associated Virus (AAV) Vectors into Infected Cells
    Yukihiko Hirai · 2023 · Nihon Ika Daigaku Igakkai Zasshi
  17. A shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice.
    Melanie D. Mark, Felix Krause, Katja Schmidtke, Mailton Vasconcelos, David Schmidt, Beyza Cansiz, Franziska Theisen, Max O. Rybarski · 2023 · Research Square
  18. O impacto da terapia gênica na saúde musculoesquelética de pacientes com hemofilia grave
    Gabriela Góes Yamaguti Hayakawa · 2022
  19. Nonclinical and Clinical Study Considerations for Biodistribution, Shedding, and Pharmacokinetics/Pharmacodynamics
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  20. rAAV Integration: Detection and Risk Assessment
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  21. Electric pulse exposure reduces AAV8 dosage required to transduce HepG2 cells
    Yizhou Yao, Robert W. Holdcraft, Susan C. Hagness, John H. Booske · 2024 · PLoS ONE
  22. Model-based dose selection for gene therapy for haemophilia B
    Elnaz Jamili, Amit Chunilal Nathwani, Vivek Dua · 2025 · Physical Sciences Reviews
  23. 33314 Model-based dose selection for gene therapy for haemophilia B
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  24. Missense and nonsense mutations and inhibitor development in patients with hemophilia A and B
    Fatemeh Karimi, Najmaldin Saki, Reyhane Khademi, Gholam Abbas Kaydani, Bijan Keikhaei · 2025 · Journal of Thrombosis and Thrombolysis
  25. Additional file 2 of The experiences of people with haemophilia and their families of gene therapy in a clinical trial setting: regaining control, the Exigency study
    Fletcher, Simon, Kathryn Jenner, Luke Pembroke, Holland, Michael, Kate Khair · 2022 · Figshare
  26. Additional file 1 of The experiences of people with haemophilia and their families of gene therapy in a clinical trial setting: regaining control, the Exigency study
    Simon J. Fletcher, Kathryn Jenner, Luke Pembroke, Holland, Michael, Kate Khair · 2022 · Figshare
  27. Additional file 6 of Human umbilical cord mesenchymal stem cell-based gene therapy for hemophilia B using scAAV-DJ/8-LP1-hFIXco transduction
    Zibin Bu, JinTu Lou, Weiqun Xu, Zhang, Lingyan, Tang, Yongmin · 2024 · Figshare
  28. Additional file 5 of Human umbilical cord mesenchymal stem cell-based gene therapy for hemophilia B using scAAV-DJ/8-LP1-hFIXco transduction
    Zibin Bu, JinTu Lou, Xu, Weiqun, Zhang, Lingyan, Tang, Yongmin · 2024 · Figshare
  29. Additional file 1 of Gene delivery to breast cancer by incorporated EpCAM targeted DARPins into AAV2
    Yafeng Lv, Zhang, Hao, Cui, Zhi, Ma, Cui-jiao, Li, Yu-ling, Lu, Hua, Wu, Hong-yan, Yang, Jian-lin, Chunyu Cao, Sun, Wen-zheng et al. · 2023 · Figshare
  30. Additional file 1 of Human umbilical cord mesenchymal stem cell-based gene therapy for hemophilia B using scAAV-DJ/8-LP1-hFIXco transduction
    Zibin Bu, Lou, Jintu, Xu, Weiqun, Zhang, Lingyan, Tang, Yongmin · 2024 · Figshare
  31. Additional file 3 of Human umbilical cord mesenchymal stem cell-based gene therapy for hemophilia B using scAAV-DJ/8-LP1-hFIXco transduction
    Zibin Bu, Lou, Jintu, Xu, Weiqun, Zhang, Lingyan, Tang, Yongmin · 2024 · Figshare
  32. Additional file 4 of Human umbilical cord mesenchymal stem cell-based gene therapy for hemophilia B using scAAV-DJ/8-LP1-hFIXco transduction
    Zibin Bu, Lou, Jintu, Weiqun Xu, Zhang, Lingyan, Tang, Yongmin · 2024 · Figshare
  33. Additional file 2 of Gene delivery to breast cancer by incorporated EpCAM targeted DARPins into AAV2
    Yafeng Lv, Zhang, Hao, Cui, Zhi, Ma, Cui-jiao, Li, Yu-ling, Lu, Hua, Wu, Hong-yan, Yang, Jian-lin, Chunyu Cao, Sun, Wen-zheng et al. · 2024 · Figshare
  34. Additional file 2 of Human umbilical cord mesenchymal stem cell-based gene therapy for hemophilia B using scAAV-DJ/8-LP1-hFIXco transduction
    Zibin Bu, Lou, Jintu, Xu, Weiqun, Zhang, Lingyan, Tang, Yongmin · 2024 · Figshare
  35. Additional file 7 of Human umbilical cord mesenchymal stem cell-based gene therapy for hemophilia B using scAAV-DJ/8-LP1-hFIXco transduction
    Zibin Bu, Lou, Jintu, Weiqun Xu, Zhang, Lingyan, Tang, Yongmin · 2024 · Figshare
  36. Balancing Promise and Peril: Hemophilia Gene Therapy Insights
    Saicharan Akula, Ester Borroni, Alessia Cottonaro, Antonia Follenzi, Simone Merlin · 2026 · IUBMB Life
  37. A Rationally Designed AAV9-DM Capsid with Minimal Liver Tropism
    Zoe C. Nabakowski, Izabella C. Jaramillo, Primrose Tanachaiwiwat, Geoffrey D. Keeler · 2026 · Cells
  38. Identification of computationally designed skeletal and cardiac promoters with high specificity across AAV delivery routes
    Nicole Zielinska, Erin L. Howard, Brenna A. Y. Stevens, Melanie M. Goens, Cici Yang, Elena S. B. Campbell, Madison E Hughes, Yanlong Pei, Dinghai Zheng, D. Benjamin Gordon et al. · 2026 · BMC Biotechnology
  39. 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)
  40. Réponses immunitaires et induction de tolérance pour la thérapie génique rAAV du muscle basée sur le ciblage des hépatocytes : induction de tolérance et mécanismes immunitaires liés à la transduction des hépatocytes
    Laurent Bartolo · 2018 · theses.fr (ABES)
  41. Adeno-associated virus in the liver : natural history of the infection and consequences in tumor development
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  42. Development of safe and efficient aav vectors for retinal gene therapy
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  43. Étude de stratégies thérapeutiques ciblant les anomalies calciques dans la Dystrophie Musculaire de Duchenne
    A. Creisméas · 2022 · theses.fr (ABES)
  44. Gene Therapy in Bleeding Disorders
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  45. Ultrasound-guided renal artery administration of angiopoietin-1 RNA therapy slows the progression of preclinical WT1 glomerular disease
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