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
来源：
GENES & DISEASES | 2024年 / 11卷 / 01期
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
  • [21] Recombinant adeno-associated virus vectors for cystic fibrosis gene therapy
    Flotte, TR
    CURRENT OPINION IN MOLECULAR THERAPEUTICS, 2001, 3 (05) : 497 - 502
  • [22] Cellular pathways of recombinant adeno-associated virus production for gene therapy
    Sha, Sha
    Maloney, Andrew J.
    Katsikis, Georgios
    Nguyen, Tam N. T.
    Neufeld, Caleb
    Wolfrum, Jacqueline
    Barone, Paul W.
    Springs, Stacy L.
    Manalis, Scott R.
    Sinskey, Anthony J.
    Braatz, Richard D.
    BIOTECHNOLOGY ADVANCES, 2021, 49
  • [23] Recombinant adeno-associated virus vector design and gene expression in the mammalian brain
    Paterna, JC
    Büeler, H
    METHODS, 2002, 28 (02) : 208 - 218
  • [24] Gene delivery via the hybrid vector of recombinant adeno-associated virus and polyethylenimine
    Hsu, Paul Y. -J.
    Yang, Ya-Wun
    EUROPEAN JOURNAL OF PHARMACEUTICAL SCIENCES, 2014, 52 : 62 - 68
  • [25] Orthopaedic gene therapy using recombinant adeno-associated virus vectors
    Ke, J.
    Zheng, L. W.
    Cheung, L. K.
    ARCHIVES OF ORAL BIOLOGY, 2011, 56 (07) : 619 - 628
  • [26] Recombinant adeno-associated virus vector for gene transfer to the transplanted rat heart
    Schirmer, Johannes M.
    Miyagi, Naoto
    Rao, Vinay P.
    Ricci, Davide
    Federspiel, Mark J.
    Kotin, Robert M.
    Russell, Stephen J.
    McGregor, Christopher G. A.
    TRANSPLANT INTERNATIONAL, 2007, 20 (06) : 550 - 557
  • [27] Clinical gene therapy using recombinant adeno-associated virus vectors
    C Mueller
    T R Flotte
    Gene Therapy, 2008, 15 : 858 - 863
  • [28] Recombinant adeno-associated virus vector: Is it ideal for gene delivery in liver transplantation?
    Yang, ZF
    Wu, XB
    Tsui, TY
    Hou, YD
    Luk, JM
    Fan, ST
    LIVER TRANSPLANTATION, 2003, 9 (04) : 411 - 420
  • [29] Modulation of the liver immune microenvironment by the adeno-associated virus serotype 8 gene therapy vector
    Carestia, Agostina
    Kim, Seok-Joo
    Horling, Franziska
    Rottensteiner, Hanspeter
    Lubich, Christian
    Reipert, Birgit M.
    Crowe, Brian A.
    Jenne, Craig N.
    MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2021, 20 : 95 - 108
  • [30] Adeno-associated virus as a delivery vector for gene therapy of human diseases
    Wang, Jiang-Hui
    Gessler, Dominic J.
    Zhan, Wei
    Gallagher, Thomas L.
    Gao, Guangping
    SIGNAL TRANSDUCTION AND TARGETED THERAPY, 2024, 9 (01)
12345