2021 / 05

DOI: 10.24075/brsmu.2021.051

REVIEW

Recombinant adeno-associated viruses as a gene delivery vehicle for the use in molecular medicine

Usman NYu1, Rebrikov DV1,2
About authors

1 Kulakov National Medical Research Center for Obstetrics, Gynecology and Perinatology, Moscow, Russia

2 Center for Precision Genome Editing and Genetic Technologies for Biomedicine, Pirogov Russian National Research Medical University, Moscow, Russia

Correspondence should be addressed: Natalia Usman
Oparina, 4, Moscow, 117997, Russia; ur.xednay@namsu.ailatan

About paper

Author contribution: NYu Usman — preparation of the manuscript; DV Rebrikov — editing of the manuscript.

Received: 2021-10-19 Accepted: 2021-10-29 Published online: 2021-10-31
|
  1. Chen W, Yao S, Wan J, Tian Y, Huang L, Wang S, et al. BBBcrossing adeno-associated virus vector: An excellent gene delivery tool for CNS disease treatment. J Control Release. 2021; 333: 129–38. DOI: 10.1016/j.jconrel.2021.03.029. PubMed PMID: 33775685.
  2. Wang D, Tai PWL, Gao G. Adeno-associated virus vector as a platform for gene therapy delivery. Nat Rev Drug Discov. 2019; 18 (5): 358–78. DOI: 10.1038/s41573-019-0012-9. PubMed PMID: 30710128.
  3. Wu Z, Yang H, Colosi P. Effect of genome size on AAV vector packaging. Mol Ther. 2010; 18 (1): 80–6. DOI: 10.1038/ mt.2009.255. Epub 2009 Nov 10. PubMed PMID: 19904234.
  4. Bass-Stringer S, Bernardo BC, May CN, Thomas CJ, Weeks KL, McMullen JR. Adeno-Associated Virus Gene Therapy: Translational Progress and Future Prospects in the Treatment of Heart Failure. Heart Lung Circ. 2018; 27 (11): 1285–300. DOI: 10.1016/j.hlc.2018.03.005. PubMed PMID: 29703647.
  5. Greenberg B, Butler J, Felker GM, Ponikowski P, Voors AA, Desai AS, Barnard D, Bouchard A, Jaski B, Lyon AR, Pogoda JM, Rudy JJ, Zsebo KM. Calcium upregulation by percutaneous administration of gene therapy in patients with cardiac disease (CUPID 2): a randomised, multinational, double-blind, placebocontrolled, phase 2b trial. Lancet. 2016; 387 (10024): 1178–86. DOI: 10.1016/S0140-6736(16)00082-9. PubMed PMID: 26803443.
  6. Evans CH, Ghivizzani SC, Robbins PD. Gene Delivery to Joints by Intra-Articular Injection. Hum Gene Ther. 2018; 29 (1): 2–14. DOI: 10.1089/hum.2017.181. PubMed PMID: 29160173.
  7. Huang L, Wan J, Wu Y, Tian Y, Yao Y, Yao S, et al. Challenges in adeno-associated virus-based treatment of central nervous system diseases through systemic injection. Life Sci. 2021; 270: 119142. DOI: 10.1016/j.lfs.2021.119142. PubMed PMID: 33524419.
  8. Mijanović O, Branković A, Borovjagin A, Butnaru DV, Bezrukov EA, Sukhanov RB, Shpichka A, Timashev P, Ulasov I. Battling Neurodegenerative Diseases with Adeno-Associated VirusBased Approaches. Viruses. 2020; 12 (4): 460. DOI: 10.3390/ v12040460. PubMed PMID: 32325732.
  9. Privolizzi R, Chu WS, Tijani M, Ng J. Viral gene therapy for paediatric neurological diseases: progress to clinical reality. Dev Med Child Neurol. 2021; 63 (9): 1019–29. DOI: 10.1111/dmcn.14885. PubMed PMID: 33834479.
  10. Nawaz W, Huang B, Xu S, Li Y, Zhu L, Yiqiao H, et al. AAVmediated in vivo CAR gene therapy for targeting human T-cell leukemia. Blood Cancer J. 2021; 11 (6): 119. DOI: 10.1038/ s41408-021-00508-1. PubMed PMID: 34162832.
  11. Bower JJ, Song L, Bastola P, Hirsch ML. Harnessing the Natural Biology of Adeno-Associated Virus to Enhance the Efficacy of Cancer Gene Therapy. Viruses. 2021; 13 (7): 1205. DOI: 10.3390/ v13071205. PubMed PMID: 34201599.
  12. Colón-Thillet R, Jerome KR, Stone D. Optimization of AAV vectors to target persistent viral reservoirs. Virol J. 2021; 18 (1): 85. DOI: 10.1186/s12985-021-01555-7. PubMed PMID: 33892762.
  13. Zhong L, Li B, Mah CS, Govindasamy L, Agbandje-McKenna M, Cooper M, et al. Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-efficiency transduction at lower doses. Proc Natl Acad Sci U S A. 2008; 105 (22): 7827–32. DOI: 10.1073/pnas.0802866105. PubMed PMID: 18511559.
  14. Kelemen RE, Mukherjee R, Cao X, Erickson SB, Zheng Y, Chatterjee A. A Precise Chemical Strategy To Alter the Receptor Specificity of the Adeno-Associated Virus. Angew Chem Int Ed Engl. 2016; 55 (36): 10645–9. DOI: 10.1002/anie.201604067. PubMed PMID: 27483453.
  15. Calcedo R, Morizono H, Wang L, McCarter R, He J, Jones D, et al. Adeno-associated virus antibody profiles in newborns, children, and adolescents. Clin Vaccine Immunol. 2011; 18 (9): 1586–8. DOI: 10.1128/CVI.05107-11. PubMed PMID: 21775517.
  16. Gadalla KKE, Vudhironarit T, Hector RD, Sinnett S, Bahey NG, Bailey MES, et al. Development of a Novel AAV Gene Therapy Cassette with Improved Safety Features and Efficacy in a Mouse Model of Rett Syndrome. Mol Ther Methods Clin Dev. 2017; 5: 180–90. DOI: 10.1016/j.omtm.2017.04.007. PubMed PMID: 28497075.
  17. Golebiowski D, van der Bom IMJ, Kwon CS, Miller AD, Petrosky K, Bradbury AM, et al. Direct Intracranial Injection of AAVrh8 Encoding Monkey β-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain. Hum Gene Ther. 2017; 28 (6): 510–22. DOI: 10.1089/hum.2016.109. PubMed PMID: 28132521.
  18. Majowicz A, Maczuga P, Kwikkers KL, van der Marel S, van Logtenstein R, Petry H, et al. Mir-142-3p target sequences reduce transgene-directed immunogenicity following intramuscular adeno-associated virus 1 vector-mediated gene delivery. J Gene Med. 2013; 15 (6–7): 219–32. DOI: 10.1002/jgm.2712. PubMed PMID: 23658149.
  19. Chamberlain K, Riyad JM, Weber T. Expressing Transgenes That Exceed the Packaging Capacity of Adeno-Associated Virus Capsids. Hum Gene Ther Methods. 2016; 27 (1): 1–12. DOI: 10.1089/hgtb.2015.140. PubMed PMID: 26757051.
  20. Kaeppel C, Beattie SG, Fronza R, van Logtenstein R, Salmon F, Schmidt S, et al. A largely random AAV integration profile after LPLD gene therapy. Nat Med. 2013; 19 (7): 889–91. DOI: 10.1038/nm.3230. PubMed PMID: 23770691.
  21. Liu M, Maurano MT, Wang H, Qi H, Song CZ, Navas PA, et al. Genomic discovery of potent chromatin insulators for human gene therapy. Nat Biotechnol. 2015; 33 (2): 198–203. DOI: 10.1038/ nbt.3062. PubMed PMID: 25580597.
  22. Clark KR, Voulgaropoulou F, Fraley DM, Johnson PR. Cell lines for the production of recombinant adeno-associated virus. Hum Gene Ther. 1995; 6 (10): 1329–41. DOI: 10.1089/hum.1995.6.101329. PubMed PMID: 8590738.
  23. Clément N, Knop DR, Byrne BJ. Large-scale adeno-associated viral vector production using a herpesvirus-based system enables manufacturing for clinical studies. Hum Gene Ther. 2009; 20 (8): 796–806. DOI: 10.1089/hum.2009.094. PubMed PMID: 19569968.
  24. Grieger JC, Soltys SM, Samulski RJ. Production of Recombinant Adeno-associated Virus Vectors Using Suspension HEK293 Cells and Continuous Harvest of Vector From the Culture Media for GMP FIX and FLT1 Clinical Vector. Mol Ther. 2016; 24 (2): 287–97. DOI: 10.1038/mt.2015.187. PubMed PMID: 26437810.
  25. Kondratov O, Marsic D, Crosson SM, Mendez-Gomez HR, Moskalenko O, Mietzsch M, et al. Direct Head-to-Head Evaluation of Recombinant Adeno-associated Viral Vectors Manufactured in Human versus Insect Cells. Mol Ther. 2017; 25 (12): 2661–75. DOI: 10.1016/j.ymthe.2017.08.003. PubMed PMID: 28890324.
  26. Nass SA, Mattingly MA, Woodcock DA, Burnham BL, Ardinger JA, Osmond SE, et al. Universal Method for the Purification of Recombinant AAV Vectors of Differing Serotypes. Mol Ther Methods Clin Dev. 2017; 9: 33–46. DOI: 10.1016/j.omtm.2017.12.004. PubMed PMID: 29349097.
  27. Available from: https://en.wikipedia.org/wiki/Alipogene_tiparvovec.
  28. Balasubramanian S, Aggarwal P, Sharma S. Lipoprotein Lipase Deficiency. In: StatPearls Treasure Island (FL): StatPearls Publishing; 2021. PubMed PMID: 32809630.
  29. Available from: https://en.wikipedia.org/wiki/Voretigene_neparvovec.
  30. Available from: https://www.askbio.com/newly-approved-spinalmuscular-atrophy-gene-therapy-zolgensma-validates-askbiogene-therapy-platform/.
  31. Available from: https://clinicaltrials.gov/ct2/results?cond=&term=adenoassociated+virus&cntry=&state=&city=&dist= retrieved on 202110-26.