Adeno-Associated Virus Gene Therapy for Liver Disease

Kattenhorn, Lisa M., Tipper, Christopher H., Stoica, Lorelei, Geraghty, Deborah S., Wright, Teresa L., Clark, K. Reed, Wadsworth, Samuel C.·2016·Human Gene Therapy

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Items 101–138 of 138

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  9. Expression Cassette Design and Regulation of Transgene Expression
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  10. Biodistribution and safety of a single rAAV3B-AAT vector for silencing and replacement of alpha-1 antitrypsin in Cynomolgus macaques
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  15. Co-opting regulation bypass repair as a gene-correction strategy for monogenic diseases
    Jingjie Hu, Rebecca A. Bourne, Barbara C. McGrath, Alice Lin, Zifei Pei, Douglas R. Cavener · 2021 · Molecular Therapy
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  17. Liver Lymphatic Dysfunction as a Driver of Fibrosis and Cirrhosis Progression
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  18. Adeno-Associated virus 8 delivers an immunomodulatory peptide to mouse liver more efficiently than to rat liver
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  19. Gene editing: a near future for the treatment of genetic kidney diseases
    Fernando Gómez-García, Miguel Ángel García-González · 2024 · Kidney International
  20. Liver Transplantation for Monogenic Metabolic Diseases Involving the Kidney
    Maurizio Salvadori, Aris Tsalouchos · 2017 · Journal of Renal and Hepatic Disorders
  21. Transplantation hépatique et futures alternatives
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  22. Generation and characterization of a new conditional mouse model of Hutchinson-Gilford Progeria Syndrome to assess disease progression upon progerin suppression and lamin A restoration
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  23. APPLICATION OF RADIOISOTOPES AND IONIZING RADIATION FOR INSECT CONTROL.
    W. Kloft, A. Suess, A. Haisch · 1971 · OSTI OAI (U.S. Department of Energy Office of Scientific and Technical Information)
  24. Gene Therapy
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  25. Role of pituitary adenylate cyclase-activating polypeptide in energy expenditure including the thermogenic response
    Daemon L. Cline · 2020
  26. Faculty Opinions recommendation of Adeno-associated virus vector as a platform for gene therapy delivery.
    Michael George Fehlings · 2020 · Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature
  27. The Alphaherpesvirus Latency Associated Promoter 2 (LAP2) Drives Strong Transgene Expression in Peripheral Tissue Depending on Administration Route and AAV Serotype
    Carola J. Maturana, Angela Chan, Jessica L. Verpeut, Esteban A. Engel · 2022 · bioRxiv (Cold Spring Harbor Laboratory)
  28. Human Hepatocyte Transduction with Adeno-Associated Virus Vector
    Zhenwei Song, Wenwei Shao, Liujiang Song, Xieolei Pei, Chengwen Li · 2022 · Methods in molecular biology
  29. Gene Therapy and Gene Editing
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  30. Lipid nanoparticle-mediated delivery of mRNA for IL-21 achieves clearance of hepatitis B virus in mouse models
    Zhongliang Shen, Shenyan Zhang, Nannan Liu, Qirong Jiang, Zixiang Gao, Weiju Hao, Qiang Deng, Jing Liu, Jiming Zhang, Youhua Xie · 2023
  31. Optimizing gene therapy for infant mice with citrullinemia type 1
    Andrea Bazo · 2023
  32. Strategies to target and genetically modify the liver sinusoid
    Lara J. Westwood, David George Le Couteur, Nicholas J. Hunt, Victoria Carroll Cogger · 2024 · Elsevier eBooks
  33. Novel AAV843 Vector-Mediated Gene Replacement Therapy Rescues Primary Hyperoxaluria Type I in Mice
    Jingjia Zhang, Ye Yin, Baowei Ji, Manqing Sun, Yuanyan Jiang, Xiaohui Wu, Xiao Xiao, Qian Shen, Xia Wu, Hong Xu · 2026 · Cells
  34. Adeno-associated virus in the liver : natural history of the infection and consequences in tumor development
    Tiziana La Bella · 2018 · theses.fr (ABES)
  35. Caractérisation de l’environnement thymique et de la thymopoïèse en conditions physiologiques et dans le contexte de stratégies thérapeutiques par ciblage direct du thymus
    Alice Machado · 2021
  36. Gene therapy for liver diseases: methods, challenges and opportunities
    Mengyao Yan, Qian Xiang · 2026 · Journal of Nanobiotechnology
  37. Inborn Errors of Metabolism
    Kayla T. Hartjes, Nanda N. Kerkar · 2026 · Gastroenterology Clinics of North America
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    Busra Cetin, Melike Aliciaslan, Ezgi Erbasan, Büşra Cesur, Salih Şanlıoğlu · 2026 · Expert Reviews in Molecular Medicine