Recombinant adeno-associated virus 8 vector in gene therapy: Opportunities and challenges

被引:14
|
作者
Zhao, Liyuan [1 ,2 ,4 ]
Yang, Zixuan [3 ]
Zheng, Minhui [3 ]
Shi, Lei [3 ]
Gu, Mengyun [3 ]
Liu, Gang [4 ]
Miao, Feng [4 ]
Chang, Yan [3 ]
Huang, Fanghua [5 ,7 ]
Tang, Naping [2 ,3 ,6 ]
机构
[1] Anhui Univ Tradit Chinese Med, Hefei 230000, Anhui, Peoples R China
[2] Yangtze Delta Pharmaceut Coll, Yangtze Delta Drug Adv Res Inst, Nantong 226133, Peoples R China
[3] Shanghai Duomirui Biotechnol Ltd, China State Inst Pharmaceut Ind, Shanghai 201203, Peoples R China
[4] InnoStar Biotech Nantong Co Ltd, Nantong 226133, Peoples R China
[5] Natl Med Prod Adm, Ctr Drug Evaluat, Beijing 100022, Peoples R China
[6] Shanghai Innostar Biotech Co Ltd, China State Inst Pharmaceut Ind, 199 Guoshoujing Rd, Shanghai 201203, Peoples R China
[7] Natl Med Prod Adm, Ctr Drug Evaluat, 128 Jianguo Rd, Beijing 100022, Peoples R China
关键词
AAV8; Adeno-associated virus; Gene therapy; Primates; Recombinant; Rodents; HEMOPHILIA-A MICE; NEUTRALIZING ANTIBODIES; EFFICIENT TRANSDUCTION; LIVER TRANSDUCTION; PERIPHERAL VEIN; RHESUS-MONKEYS; VIRAL VECTORS; MOUSE MODEL; AAV VECTOR; SEROTYPE-8;
D O I
10.1016/j.gendis.2023.02.010
中图分类号
Q5 [生物化学]; Q7 [分子生物学];
学科分类号
071010 ; 081704 ;
摘要
In recent years, significant breakthroughs have been made in the field of gene ther-apy. Adeno-associated virus (AAV) is one of the most promising gene therapy vectors and a powerful tool for delivering the gene of interest. Among the AAV vectors, AAV serotype 8 (AAV8) has attracted much attention for its efficient and stable gene transfection into specific tissues. Currently, recombinant AAV8 has been widely used in gene therapy research on a va-riety of diseases, including genetic diseases, cancers, autoimmune diseases, and viral diseases. This paper reviewed the applications and challenges of using AAV8 as a vector for gene ther-apy, with the aim of providing a valuable resource for those pursuing the application of viral vectors in gene therapy. 2023 The Authors. Publishing services by Elsevier B.V. on behalf of KeAi Communications Co., Ltd. This is an open access article under the CC BY-NC-ND license (http://creativecommons. org/licenses/by-nc-nd/4.0/).
引用
收藏
页码:283 / 293
页数:11
相关论文
共 50 条
  • [41] Adeno-associated virus vectors for human gene therapy
    Haifeng Chen
    World Journal of Medical Genetics, 2015, (03) : 28 - 45
  • [42] Adeno-Associated Virus-Mediated Gene Therapy
    Zaman, Hassan
    Khan, Aakif
    Khan, Khalid
    Toheed, Shazma
    Abdullah, Muhammad
    Zeeshan, Hafiz Muhammad
    Hameed, Abdul
    Umar, Muhammad
    Shahid, Muhammad
    Malik, Kausar
    Afzal, Samia
    CRITICAL REVIEWS IN EUKARYOTIC GENE EXPRESSION, 2023, 33 (06): : 87 - 100
  • [43] Hot topics in adeno-associated virus as a gene transfer vector
    Na Zhao
    De-Pei Liu
    Chih-Chuan Liang
    Molecular Biotechnology, 2001, 19 : 229 - 237
  • [44] Hot topics in adeno-associated virus as a gene transfer vector
    Zhao, N
    Liu, DP
    Liang, CC
    MOLECULAR BIOTECHNOLOGY, 2001, 19 (03) : 229 - 237
  • [45] Adeno-associated virus as a gene therapy vector: Vector development, production and clinical applications
    Grieger, JC
    Samulski, RJ
    GENE THERAPY AND GENE DELIVERY SYSTEMS, 2005, 99 : 119 - 145
  • [46] Cardiac gene therapy with adeno-associated virus-based vectors
    Chamberlain, Kyle
    Riyad, Jalish M.
    Weber, Thomas
    CURRENT OPINION IN CARDIOLOGY, 2017, 32 (03) : 275 - 282
  • [47] hEPO transfer and expression by a recombinant adeno-associated virus vector
    Wu, ZJ
    Wu, XB
    Wang, H
    Lu, B
    Dong, XY
    Hou, YD
    ACTA BIOCHIMICA ET BIOPHYSICA SINICA, 2002, 34 (02) : 176 - 180
  • [48] Bioanalysis of adeno-associated virus gene therapy therapeutics: regulatory expectations
    Gorovits, Boris
    Marshall, Jean-Claude
    Smith, Justin
    Whiteley, Laurence O.
    Neubert, Hendrik
    BIOANALYSIS, 2019, 11 (21) : 2011 - 2024
  • [49] Synthetically Engineered Adeno-Associated Virus for Efficient, Safe, and Versatile Gene Therapy Applications
    Lugin, Margaret L.
    Lee, Rebecca T.
    Kwon, Young Jik
    ACS NANO, 2020, 14 (11) : 14262 - 14283
  • [50] Adeno-Associated Virus Vectors: Principles, Practices, and Prospects in Gene Therapy
    Zwi-Dantsis, Limor
    Mohamed, Saira
    Massaro, Giulia
    Moeendarbary, Emad
    VIRUSES-BASEL, 2025, 17 (02):