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

页码：283 / 293

页数：11

共 50 条

[41] Mechanisms of antigen presentation in recombinant adeno-associated virus (rAAV) vector mediated vaccination and gene therapy
Xu, Dan
Velazquez, Victoria
Schaffer, David
Kaspari, Brian
Walker, Christopher
JOURNAL OF IMMUNOLOGY, 2010, 184
[42] 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
[43] Adeno-Associated Virus: The Naturally Occurring Virus Versus the Recombinant Vector
Srivastava, Arun
HUMAN GENE THERAPY, 2016, 27 (01) : 1 - 6
[44] 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
[45] My Pathway to Adeno-Associated Virus and Adeno-Associated Virus Gene Therapy: A Personal Perspective
Carter, Barrie J.
HUMAN GENE THERAPY, 2020, 31 (9-10) : 494 - 498
[46] Recombinant Adeno-Associated Virus Vector Tropism in Human Retina
Andrzejewski, Slawomir
Moyle, Peter M.
Stringer, Brett W.
Steel, Jason
Layton, Christopher J.
MOLECULAR THERAPY, 2020, 28 (04) : 254 - 254
[47] Recombinant adeno-associated virus (rAAV)-based gene therapy for Lafora disease
Zafra-Puerta, L.
Burgos, D. F.
Iglesias-Cabeza, N.
Sanchez-Martin, G.
Sanchez, M. P.
Serratosa, J. M.
HUMAN GENE THERAPY, 2024, 35 (3-4) : A188 - A188
[48] Gene Therapy Progress and Prospects: Recombinant adeno-associated virus (rAAV) vectors
T R Flotte
Gene Therapy, 2004, 11 : 805 - 810
[49] Transfer of the feline erythropoietin gene to cats using a recombinant adeno-associated virus vector
C J Beall
A J Phipps
L E Mathes
P Stromberg
P R Johnson
Gene Therapy, 2000, 7 : 534 - 539
[50] Targeted gene expression in the oligodendrocytes using a recombinant adeno-associated virus vector.
Chen, H
McCarty, DM
Bruce, AT
Suzuki, K
Suzuki, K
JOURNAL OF NEUROPATHOLOGY AND EXPERIMENTAL NEUROLOGY, 1998, 57 (05): : 505 - 505

← 1 2 3 4 5 →