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

Liyuan Zhao; Zixuan Yang; Minhui Zheng; Lei Shi; Mengyun Gu; Gang Liu; Feng Miao; Yan Chang; Fanghua Huang; Naping Tang

doi:10.1016/j.gendis.2023.02.010

您当前的位置：

首页 >

文章列表页 >

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

Review Articles | 更新时间：2024-02-04

- Recombinant adeno-associated virus 8 vector in gene therapy: Opportunities and challenges
- Recombinant adeno-associated virus 8 vector in gene therapy: Opportunities and challenges
- Genes & Diseases 2024年1卷第1期页码：283-293
- 作者机构：
  
  1. Anhui University of Traditional Chinese Medicine,Anhui,Hefei,China,230000
  2. Yangtze Delta Drug Advanced Research Institute, Yangtze Delta Pharmaceutical College,Jiangsu,Nantong,China,226133
  3. InnoStar Bio-tech Nantong Co., Ltd.,Jiangsu,Nantong,China,226133
  4. Shanghai Innostar Bio-Technology Co., Ltd, China State Institute of Pharmaceutical Industry,Shanghai,China,201203
  5. Center for Drug Evaluation, National Medical Products Administration,Beijing,China,100022
- 作者简介：
- 基金信息：
- DOI：10.1016/j.gendis.2023.02.010
  中图分类号：
- 纸质出版日期：2024，
  
  网络出版日期：2024-1-1，
扫描看全文
Recombinant adeno-associated virus 8 vector in gene therapy: Opportunities and challenges[J]. Genes & Diseases, 2024,1(1):283-293.

Liyuan Zhao, Zixuan Yang, Minhui Zheng, et al. Recombinant adeno-associated virus 8 vector in gene therapy: Opportunities and challenges[J]. Genes & Diseases, 2024,1(1):283-293.
Recombinant adeno-associated virus 8 vector in gene therapy: Opportunities and challenges[J]. Genes & Diseases, 2024,1(1):283-293. DOI： 10.1016/j.gendis.2023.02.010.

Liyuan Zhao, Zixuan Yang, Minhui Zheng, et al. Recombinant adeno-associated virus 8 vector in gene therapy: Opportunities and challenges[J]. Genes & Diseases, 2024,1(1):283-293. DOI： 10.1016/j.gendis.2023.02.010.

摘要

Abstract

In recent years

significant breakthroughs have been made in the field of gene therapy. 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 variety 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 therapy

with the aim of providing a valuable resource for those pursuing the application of viral vectors in gene therapy.

关键词

Keywords

AAV8Adeno-associated virusGene therapyPrimatesRecombinantRodents

references

1 S.L. Ginn, A.K. Amaya, I.E. Alexander, et al.Gene therapy clinical trials worldwide to 2017: an update J Gene Med, 20 (5) (2018), Article e3015

2 A. Fischer, S. Hacein-Bey-AbinaGene therapy for severe combined immunodeficiencies and beyond J Exp Med, 217 (2) (2020), Article e20190607

3 S.E. Raper, N. Chirmule, F.S. Lee, et al.Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer Mol Genet Metabol, 80 (1–2) (2003), pp. 148-158

4 S. Hacein-Bey-Abina, C. Von Kalle, M. Schmidt, et al.LMO2- associated clonal T cell proliferation in two patients after gene therapy for SCID-X1 Science, 302 (5644) (2003), pp. 415-419

5 L. Wang, R. Calcedo, T.C. Nichols, et al.Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy Blood, 105 (8) (2005), pp. 3079-3086

6 H.J. Nam, M.D. Lane, E. Padron, et al.Structure of adeno-associated virus serotype 8, a gene therapy vector J Virol, 81 (22) (2007), pp. 12260-12271

7 B. Akache, D. Grimm, K. Pandey, et al.The 37/67-kilodalton laminin receptor is a receptor for adeno-associated virus serotypes 8, 2, 3, and 9 J Virol, 80 (19) (2006), pp. 9831-9836

8 M. Mietzsch, C. Barnes, J.A. Hull, et al.Comparative analysis of the capsid structures of AAVrh.10, AAVrh.39, and AAV8 J Virol, 94 (6) (2020) :e01769-e01719

9 I.P. Seitz, S. Michalakis, B. Wilhelm, et al.Superior retinal gene transfer and biodistribution profile of subretinal versus intravitreal delivery of AAV8 in nonhuman Primates Invest Ophthalmol Vis Sci, 58 (13) (2017), pp. 5792-5801

10 V.P. Chen, Y. Gao, L. Geng, et al.Systemic safety of a recombinant AAV8 vector for human cocaine hydrolase gene therapy: a good laboratory practice preclinical study in mice Hum Gene Ther, 31 (1–2) (2020), pp. 70-79

11 J.A. Greig, H. Peng, J. Ohlstein, et al.Intramuscular injection of AAV8 in mice and macaques is associated with substantial hepatic targeting and transgene expression PLoS One, 9 (11) (2014), Article e112268

12 G.P. Gao, M.R. Alvira, L. Wang, et al.Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy Proc Natl Acad Sci U S A, 99 (18) (2002), pp. 11854-11859

13 L. Wang, P. Bell, J. Lin, et al.AAV8-mediated hepatic gene transfer in infant Rhesus monkeys (Macaca mulatta) Mol Ther, 19 (11) (2011), pp. 2012-2020

14 H. Nakai, S. Fuess, T.A. Storm, et al.Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice J Virol, 79 (1) (2005), pp. 214-224

15 W. Shao, X. Pei, C. Cui, et al.Superior human hepatocyte transduction with adeno-associated virus vector serotype 7 Gene Ther, 26 (12) (2019), pp. 504-514

16 R. Sarkar, R. Tetreault, G. Gao, et al.Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype Blood, 103 (4) (2004), pp. 1253-1260

17 J.H. Mao, Y. Shen, Q. Wang, et al.Optimized AAV package and experimental application of recombinant AAV8/hFⅧ for gene therapy on hemophilia A mice Zhonghua Xue Ye Xue Za Zhi, 41 (1) (2020), pp. 34-39

18 X. Pan, Y. Yue, K. Zhang, et al.Long-term robust myocardial transduction of the dog heart from a peripheral vein by adeno-associated virus serotype-8 Hum Gene Ther, 24 (6) (2013), pp. 584-594

19 Z. Wang, T. Zhu, C. Qiao, et al.Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart Nat Biotechnol, 23 (3) (2005), pp. 321-328

20 A.Y. Wang, P.D. Peng, A. Ehrhardt, et al.Comparison of adenoviral and adeno-associated viral vectors for pancreatic gene delivery in vivo Hum Gene Ther, 15 (4) (2004), pp. 405-413

21 A. Ramzy, E. Tudurí, M.M. Glavas, et al.AAV8 Ins1-Cre can produce efficient β-cell recombination but requires consideration of off-target effects Sci Rep, 10 (2020), Article 10518

22 D.F. Aschauer, S. Kreuz, S. RumpelAnalysis of transduction efficiency, tropism and axonal transport of AAV serotypes 1, 2, 5, 6, 8 and 9 in the mouse brain PLoS One, 8 (9) (2013), Article e76310

23 J.I. Ayers, S. Fromholt, O. Sinyavskaya, et al.Widespread and efficient transduction of spinal cord and brain following neonatal AAV injection and potential disease-modifying effect in ALS mice Mol Ther, 23 (1) (2015), pp. 53-62

24 A.M. Davidoff, C.C. Ng, J. Zhou, et al.Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway Blood, 102 (2) (2003), pp. 480-488

25 J.B. Nietupski, G.D. Hurlbut, R.J. Ziegler, et al.Systemic administration of AAV8-α-galactosidase A induces humoral tolerance in nonhuman Primates despite low hepatic expression Mol Ther, 19 (11) (2011), pp. 1999-2011

26 A.E. Vozenilek, C.M.R. Blackburn, R.M. Schilke, et al.AAV8-mediated overexpression of mPCSK9 in liver differs between male and female mice Atherosclerosis, 278 (2018), pp. 66-72

27 A. Pañeda, L. Vanrell, I. Mauleon, et al.Effect of adeno-associated virus serotype and genomic structure on liver transduction and biodistribution in mice of both genders Hum Gene Ther, 20 (8) (2009), pp. 908-917

28 S.J. Aronson, P. Veron, F. Collaud, et al.Prevalence and relevance of pre-existing anti-adeno-associated virus immunity in the context of gene therapy for crigler-najjar syndrome Hum Gene Ther, 30 (10) (2019), pp. 1297-1305

29 A. Kruzik, D. Fetahagic, B. Hartlieb, et al.Prevalence of anti-adeno-associated virus immune responses in international cohorts of healthy donors Mol Ther Methods Clin Dev, 14 (2019), pp. 126-133

30 A. Kruzik, H. Koppensteiner, D. Fetahagic, et al.Detection of biologically relevant low-titer neutralizing antibodies against adeno-associated virus require sensitive in vitro assays Hum Gene Ther Methods, 30 (2) (2019), pp. 35-43

31 V. Louis Jeune, J.A. Joergensen, R.J. Hajjar, et al.Pre-existing anti-adeno-associated virus antibodies as a challenge in AAV gene therapy Hum Gene Ther Methods, 24 (2) (2013), pp. 59-67

32 J. Mimuro, H. Mizukami, M. Shima, et al.The prevalence of neutralizing antibodies against adeno-associated virus capsids is reduced in young Japanese individuals J Med Virol, 86 (11) (2014), pp. 1990-1997

33 S. Boutin, V. Monteilhet, P. Veron, et al.Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors Hum Gene Ther, 21 (6) (2010), pp. 704-712

34 S. Stanford, R. Pink, D. Creagh, et al.Adenovirus-associated antibodies in UK cohort of hemophilia patients: a seroprevalence study of the presence of adenovirus-associated virus vector-serotypes AAV5 and AAV8 neutralizing activity and antibodies in patients with hemophilia A Res Pract Thromb Haemost, 3 (2) (2019), pp. 261-267

35 L. Sun, L. Tu, G. Gao, et al.Assessment of a passive immunity mouse model to quantitatively analyze the impact of neutralizing antibodies on adeno-associated virus-mediated gene transfer J Immunol Methods, 387 (1–2) (2013), pp. 114-120

36 R. Watano, T. Ohmori, S. Hishikawa, et al.Utility of microminipigs for evaluating liver-mediated gene expression in the presence of neutralizing antibody against vector capsid Gene Ther, 27 (9) (2020), pp. 427-434

37 J. Sun, W. Shao, X. Chen, et al.An observational study from long-term AAV re-administration in two hemophilia dogs Mol Ther Methods Clin Dev, 10 (2018), pp. 257-267

38 J. Lengler, S. Coulibaly, B. Gruber, et al.Development of an in vitro biopotency assay for an AAV8 hemophilia B gene therapy vector suitable for clinical product release Mol Ther Methods Clin Dev, 17 (2020), pp. 581-588

39 L. Wang, I. Muthuramu, S. Somanathan, et al.Developing a second-generation clinical candidate AAV vector for gene therapy of familial hypercholesterolemia Mol Ther Methods Clin Dev, 22 (2021), pp. 1-10

40 S.O. Han, D. Gheorghiu, S. Li, et al.Minimum effective dose to achieve biochemical correction with adeno-associated virus vector-mediated gene therapy in mice with Pompe disease Hum Gene Ther, 33 (9–10) (2022), pp. 492-498

41 M. Chen, K. Maeng, A. Nawab, et al.Efficient gene delivery and expression in pancreas and pancreatic tumors by capsid-optimized AAV8 vectors Hum Gene Ther Methods, 28 (1) (2017), pp. 49-59

42 R.M. Tenney, C.L. Bell, J.M. WilsonAAV8 capsid variable regions at the two-fold symmetry axis contribute to high liver transduction by mediating nuclear entry and capsid uncoating Virology, 454–455 (2014), pp. 227-236

43 H. Jiang, D. Lillicrap, S. Patarroyo-White, et al.Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs Blood, 108 (1) (2006), pp. 107-115

44 W. Shao, J. Sun, X. Chen, et al.Chimeric mice engrafted with canine hepatocytes exhibits similar AAV transduction efficiency to hemophilia B dog Front Pharmacol, 13 (2022), Article 815317

45 A.C. Nathwani, J.T. Gray, J. McIntosh, et al.Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates Blood, 109 (4) (2007), pp. 1414-1421

46 B. Zhou, H. Qiao, L. Tong, et al.Apoptosis-inducing effect of recombinant adeno-associated virus serotype 8 with TRAIL gene on human colon cancer cell line Chin J Gen Surg (4) (2006), pp. 281-283

47 C.H. Lo, C.M. Chang, S.W. Tang, et al.Differential antitumor effect of interleukin-12 family cytokines on orthotopic hepatocellular carcinoma J Gene Med, 12 (5) (2010), pp. 423-434

48 C.M. Chang, C.H. Lo, Y.M. Shih, et al.Treatment of hepatocellular carcinoma with adeno-associated virus encoding interleukin-15 superagonist Hum Gene Ther, 21 (5) (2010), pp. 611-621

49 L. Yin, G.D. Keeler, Y. Zhang, et al.AAV3-miRNA vectors for growth suppression of human hepatocellular carcinoma cells in vitro and human liver tumors in a murine xenograft model in vivo Gene Ther, 28 (7–8) (2021), pp. 422-434

50 W. Jiang, H. Li, X. Liu, et al.Precise and efficient silencing of mutant KrasG12D by CRISPR-CasRx controls pancreatic cancer progression Theranostics, 10 (25) (2020), pp. 11507-11519

51 F. Kósa, P. Kunovszki, J. Gimesi-Országh, et al.High risk of depression, anxiety, and an unfavorable complex comorbidity profile is associated with SLE: a nationwide patient-level study Arthritis Res Ther, 24 (2022), p. 116

52 P. Yang, X. Zhang, S. Chen, et al.A novel serum tsRNA for diagnosis and prediction of nephritis in SLE Front Immunol, 12 (2021), Article 735105

53 X. Ye, T. Zhu, S. Bastacky, et al.Prevention and reversal of lupus in NZB/NZW mice by costimulatory blockade with adeno-associated virus-mediated gene transfer Arthritis Rheum, 52 (12) (2005), pp. 3975-3986

54 J.P. Casazza, E.M. Cale, S. Narpala, et al.Safety and tolerability of AAV8 delivery of a broadly neutralizing antibody in adults living with HIV: a phase 1, dose-escalation trial Nat Med, 28 (5) (2022), pp. 1022-1030

55 S.U. Gan, M. Notaridou, Z.Y. Fu, et al.Correction of murine diabetic hyperglycaemia with a single systemic administration of an AAV2/8 vector containing a novel codon optimized human insulin gene Curr Gene Ther, 16 (1) (2016), pp. 65-72

56 S.U. Gan, Z. Fu, K.C. Sia, et al.Development of a liver-specific Tet-off AAV8 vector for improved safety of insulin gene therapy for diabetes J Gene Med, 21 (1) (2019), Article e3067

57 F. Manzoor, M.C. Johnson, C. Li, et al.β-cell-specific IL-35 therapy suppresses ongoing autoimmune diabetes in NOD mice Eur J Immunol, 47 (1) (2017), pp. 144-154

58 A. PhilippidisFourth boy dies in clinical trial of Astellas' AT132 Hum Gene Ther, 32 (19–20) (2021), pp. 1008-1010

59 Astellas’ Pompe disease gene therapy placed on hold by FDA Cell Gene Therapy (2022) https://www.fiercebiotech.com/biotech/astellas-gene-therapy-strategy-faces-fresh-setback-fda-puts-pompe-disease-drug-hold

60 G.N. Nguyen, J.K. Everett, S. Kafle, et al.A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells Nat Biotechnol, 39 (1) (2021), pp. 47-55

61 H.R. Kang, M. Gjorgjieva, S.N. Smith, et al.Pathogenesis of hepatic tumors following gene therapy in murine and canine models of glycogen storage disease Mol Ther Methods Clin Dev, 15 (2019), pp. 383-391

62 J.A. Greig, M.P. Limberis, P. Bell, et al.Non-clinical study examining AAV8.TBG.hLDLR vector-associated toxicity in chow-fed wild-type and LDLR+/− Rhesus macaques Hum Gene Ther Clin Dev, 28 (1) (2017), pp. 39-50

63 K.J. Ho, C.E. Bass, A.H.K. Kroemer, et al.Optimized adeno-associated virus 8 produces hepatocyte-specific Cre-mediated recombination without toxicity or affecting liver regeneration Am J Physiol Gastrointest Liver Physiol, 295 (2) (2008), pp. G412-G419

64 J.A. Greig, M.P. Limberis, P. Bell, et al.Nonclinical pharmacology/toxicology study of AAV8.TBG.mLDLR and AAV8.TBG.hLDLR in a mouse model of homozygous familial hypercholesterolemia Hum Gene Ther Clin Dev, 28 (1) (2017), pp. 28-38

65 M.L.D. Broekman, L.A. Comer, B.T. Hyman, et al.Adeno-associated virus vectors serotyped with AAV8 capsid are more efficient than AAV-1 or-2 serotypes for widespread gene delivery to the neonatal mouse brain Neuroscience, 138 (2) (2006), pp. 501-510

66 D.A. Gross, N. Tedesco, C. Leborgne, et al.Overcoming the challenges imposed by humoral immunity to AAV vectors to achieve safe and efficient gene transfer in seropositive patients Front Immunol, 13 (2022), Article 857276

67 G.D. Hurlbut, R.J. Ziegler, J.B. Nietupski, et al.Preexisting immunity and low expression in primates highlight translational challenges for liver-directed AAV8-mediated gene therapy Mol Ther, 18 (11) (2010), pp. 1983-1994

68 B. Liu, Z. Li, S. Huang, et al.AAV-containing exosomes as a novel vector for improved gene delivery to lung cancer cells Front Cell Dev Biol, 9 (2021), Article 707607

查看原文

浏览量

下载量

CSCD

文章被引用时，请邮件提醒。

提交

工具集

关联资源

Clinical applications of the CRISPR/Cas9 genome-editing system: Delivery options and challenges in precision medicine

Prolonged control of insulin-dependent diabetes via intramuscular expression of plasmid-encoded single-strand insulin analogue

Gene therapy and genome editing for primary immunodeficiency diseases

Allotopic expression of mitochondrial genes: Basic strategy and progress

AAV6 as an effective gene delivery vector for prolonged transgene expression in intervertebral disc cells in vivo