Adeno-associated viral vector-mediated immune responses: Understanding barriers to gene delivery

被引:110
|
作者
Nidetz, Natalie F. [1 ]
McGee, Michael C. [1 ]
Tse, Longping V. [2 ]
Li, Chengwen [3 ]
Cong, Le [4 ,5 ]
Li, Yunxing [6 ]
Huang, Weishan [1 ,7 ]
机构
[1] Louisiana State Univ, Sch Vet Med, Dept Pathobiol Sci, Baton Rouge, LA 70803 USA
[2] Univ N Carolina, Sch Publ Hlth, Dept Epidemiol, Chapel Hill, NC 27599 USA
[3] Univ N Carolina, Gene Therapy Ctr, Chapel Hill, NC 27599 USA
[4] Stanford Univ, Sch Med, Dept Pathol, Stanford, CA 94305 USA
[5] Stanford Univ, Sch Med, Dept Genet, Stanford, CA 94305 USA
[6] Cornell Univ, Dept Mol Med, Ithaca, NY 14853 USA
[7] Cornell Univ, Dept Microbiol & Immunol, Ithaca, NY 14853 USA
基金
美国国家卫生研究院;
关键词
Adeno-associated virus; Gene therapy; Pre-existing immunity; Vaccination; Immune tolerance; Cytotoxic lymphocytes; REGULATORY T-CELLS; CAPSID ANTIGEN PRESENTATION; HUMAN-IMMUNODEFICIENCY-VIRUS; LONG-TERM SAFETY; AAV-FACTOR-IX; IN-VIVO; NEUTRALIZING ANTIBODIES; HUMORAL IMMUNITY; SKELETAL-MUSCLE; CLINICAL-TRIAL;
D O I
10.1016/j.pharmthera.2019.107453
中图分类号
R9 [药学];
学科分类号
1007 ;
摘要
Adeno-associated viral (MV) vectors have emerged as the leading gene delivery platform for gene therapy and vaccination. Three AAV-based gene therapy drugs, Glybera, LUXTURNA, and ZOLGENSMA were approved between 2012 and 2019 by the European Medicines Agency and the United States Food and Drug Administration as treatments for genetic diseases hereditary lipoprotein lipase deficiency (LPLD), inherited retinal disease (IRD), and spinal muscular atrophy (SMA), respectively. Despite these therapeutic successes, clinical trials have demonstrated that host anti-viral immune responses can prevent the long-term gene expression of AAV vector-encoded genes. Therefore, it is critical that we understand the complex relationship between AAV vectors and the host immune response. This knowledge could allow for the rational design of optimized gene transfer vectors capable of either subverting host immune responses in the context of gene therapy applications, or stimulating desirable immune responses that generate protective immunity in vaccine applications to AAV vector-encoded antigens. This review provides an overview of our current understanding of the AAV-induced immune response and discusses potential strategies by which these responses can be manipulated to improve AAV vector-mediated gene transfer. (C) 2019 The Author(s). Published by Elsevier Inc.
引用
收藏
页数:13
相关论文
共 50 条
  • [21] Adeno-associated virus vector-mediated transduction in the cat brain
    Vite, CH
    Passini, MA
    Haskins, ME
    Wolfe, JH
    GENE THERAPY, 2003, 10 (22) : 1874 - 1881
  • [22] Optimization of adeno-associated viral vector-mediated transduction of the corticospinal tract: comparison of four promoters
    Bart Nieuwenhuis
    Barbara Haenzi
    Sam Hilton
    Alejandro Carnicer-Lombarte
    Barbara Hobo
    Joost Verhaagen
    James W. Fawcett
    Gene Therapy, 2021, 28 : 56 - 74
  • [23] Adeno-associated virus vector-mediated triple gene transfer of dopamine synthetic enzymes
    Fan, DS
    Shen, Y
    Kang, DX
    Nakano, I
    Ozawa, K
    CHINESE MEDICAL JOURNAL, 2001, 114 (12) : 1276 - 1279
  • [24] Characteristics and advantages of adeno-associated virus vector-mediated gene therapy for neurodegenerative diseases
    Qu, Yuan
    Liu, Yi
    Noor, Ahmed Fayyaz
    Tran, Johnathan
    Li, Rui
    NEURAL REGENERATION RESEARCH, 2019, 14 (06) : 931 - 938
  • [25] Barriers to Non-Viral Vector-Mediated Gene Delivery in the Nervous System
    Perez-Martinez, Francisco C.
    Guerra, Javier
    Posadas, Inmaculada
    Cena, Valentin
    PHARMACEUTICAL RESEARCH, 2011, 28 (08) : 1843 - 1858
  • [26] Barriers to Non-Viral Vector-Mediated Gene Delivery in the Nervous System
    Francisco C. Pérez-Martínez
    Javier Guerra
    Inmaculada Posadas
    Valentín Ceña
    Pharmaceutical Research, 2011, 28 : 1843 - 1858
  • [27] Adeno-associated viral vector-mediated preprosomatostatin expression suppresses induced seizures in kindled rats
    Natarajan, Gowri
    Leibowitz, Jeffrey A.
    Zhou, Junli
    Zhao, Yang
    McElroy, Jessica A.
    King, Michael A.
    Ormerod, Brandi K.
    Carney, Paul R.
    EPILEPSY RESEARCH, 2017, 130 : 81 - 92
  • [28] Optimization of adeno-associated viral vector-mediated transduction of the corticospinal tract: comparison of four promoters
    Nieuwenhuis, Bart
    Haenzi, Barbara
    Hilton, Sam
    Carnicer-Lombarte, Alejandro
    Hobo, Barbara
    Verhaagen, Joost
    Fawcett, James W.
    GENE THERAPY, 2021, 28 (1-2) : 56 - 74
  • [29] Optimization of Adeno-Associated Viral Vector-Mediated Transduction of the Retina: Comparison of Five Short Promoters
    Nieuwenhuis, Bart
    Laperrousaz, Elise
    Verhaagen, Joost
    Fawcett, James W.
    Martin, Keith R.
    Osborne, Andrew
    MOLECULAR THERAPY, 2021, 29 (04) : 148 - 148
  • [30] Adeno-associated virus vector-mediated gene transfer into anterior horn cells.
    Acsadi, G
    Yang, H
    Anguelov, R
    Toht, G
    Wang, Y
    Shy, ME
    AMERICAN JOURNAL OF HUMAN GENETICS, 2001, 69 (04) : 679 - 679