Application of gene therapy with viral vectors in infectious and noninfectious diseases

Document Type : Systematic Review

Authors
S. Saeb 1
M. Ravanshad 2
1 1. Qaen School of Nursing and Midwifery, Birjand University of Medical Sciences, Birjand, Iran2. Department of Virology, Faculty of Medical Sciences, Tarbiat Modares University, Tehran, Iran
2 Department of Virology, Faculty of Medical Sciences, Tarbiat Modares University, Tehran, Iran
Abstract
Virus-based vectors, also called viral vectors are being studied in the field of gene therapy because of their innate biological and structural properties. Their ability to protect and specific delivery of genetic elements into the cells, promising results in the pre-clinical studies, going through clinical trials and approval of some products as a therapeutic option in some cancers introduce viral vectors as a powerful tool in the field of gene therapy. In this review, first an introduction about viral vectors is presented and then some studies about application of viral vectors in various fields of human diseases such as prevention and treatment are briefly discussed.

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1. Friedmann T, Roblin R. Gene therapy for human genetic disease? Science. 1972;175(4025):949-55.
2. Lundstrom K. Gene therapy applications of viral vectors. Technology in cancer research & treatment. 2004;3(5):467-77.
3. Lentz TB, Gray SJ, Samulski RJ. Viral vectors for gene delivery to the central nervous system. Neurobiol Dis. 2012;48(2):179-88.
4. Lundstrom K. Viral Vectors in Gene Therapy. Diseases. 2018;6(2):42.
5. van Riel D, de Wit E. Next-generation vaccine platforms for COVID-19. Nature Materials. 2020;19(8):810-2.
6. [Available from: https://www.who.int/publications/m/item/draft-landscape-of-covid-19-candidate-vaccines.
7. Voysey M, Clemens SAC, Madhi SA, Weckx LY, Folegatti PM, Aley PK, et al. Safety and efficacy of the ChAdOx1 nCoV-19 vaccine (AZD1222) against SARS-CoV-2: an interim analysis of four randomised controlled trials in Brazil, South Africa, and the UK. Lancet. 2021;397(10269):99-111.
8. Jones I, Roy P. Sputnik V COVID-19 vaccine candidate appears safe and effective. Lancet. 2021;397(10275):642-3.
9. Wu S, Zhong G, Zhang J, Shuai L, Zhang Z, Wen Z, et al. A single dose of an adenovirus-vectored vaccine provides protection against SARS-CoV-2 challenge. Nature Communications. 2020;11(1):4081.
10. Ringpis G-EE, Shimizu S, Arokium H, Camba-Colón J, Carroll MV, Cortado R, et al. Engineering HIV-1-Resistant T-Cells from Short-Hairpin RNA-Expressing Hematopoietic Stem/Progenitor Cells in Humanized BLT Mice. PLOS ONE. 2013;7(12):e53492.
11. Qin XF, An DS, Chen IS, Baltimore D. Inhibiting HIV-1 infection in human T cells by lentiviral-mediated delivery of small interfering RNA against CCR5. Proc Natl Acad Sci U S A. 2003;100(1):183-8.
12. [Available from: https://clinicaltrials.gov/ct2/show/NCT01734850?cond=NCT01734850&draw=2&rank=1.
13. Clarke DK, Cooper D, Egan MA, Hendry RM, Parks CL, Udem SA. Recombinant vesicular stomatitis virus as an HIV-1 vaccine vector. Springer seminars in immunopathology. 2006;28(3):239-53.
14. van den Berg FT, Makoah NA, Ali SA, Scott TA, Mapengo RE, Mutsvunguma LZ, et al. AAV-Mediated Expression of Broadly Neutralizing and Vaccine-like Antibodies Targeting the HIV-1 Envelope V2 Region. Molecular Therapy - Methods & Clinical Development. 2019;14:100-12.
15. Brady JM, Baltimore D, Balazs AB. Antibody gene transfer with adeno‐associated viral vectors as a method for HIV prevention. Immunological reviews. 2017;275(1):324-33.
16. Delviks-Frankenberry KA, Ackerman D, Timberlake ND, Hamscher M, Nikolaitchik OA, Hu WS, et al. Development of Lentiviral Vectors for HIV-1 Gene Therapy with Vif-Resistant APOBEC3G. Molecular therapy Nucleic acids. 2019;18:1023-38.
17. Saeb S, Ravanshad M, Pourkarim MR, Daouad F, Baesi K, Rohr O, et al. Brain HIV-1 latently-infected reservoirs targeted by the suicide gene strategy. Virology journal. 2021;18(1):1-10.
18. Huelsmann PM, Hofmann AD, Knoepfel SA, Popp J, Rauch P, Di Giallonardo F, et al. A suicide gene approach using the human pro-apoptotic protein tBid inhibits HIV-1 replication. BMC biotechnology. 2011;11:4.
19. Bishnoi S, Tiwari R, Gupta S, Byrareddy SN, Nayak D. Oncotargeting by Vesicular Stomatitis Virus (VSV): Advances in Cancer Therapy. Viruses. 2018;10(2).
20. Chulpanova DS, Solovyeva VV, Kitaeva KV, Dunham SP, Khaiboullina SF, Rizvanov AA. Recombinant Viruses for Cancer Therapy. Biomedicines. 2018;6(4):94.
21. Lykken EA, Shyng C, Edwards RJ, Rozenberg A, Gray SJ. Recent progress and considerations for AAV gene therapies targeting the central nervous system. Journal of Neurodevelopmental Disorders. 2018;10(1):16.
22. Daya S, Berns KI. Gene therapy using adeno-associated virus vectors. Clinical microbiology reviews. 2008;21(4):583-93.
23. Deverman BE, Ravina BM, Bankiewicz KS, Paul SM, Sah DWY. Gene therapy for neurological disorders: progress and prospects. Nature Reviews Drug Discovery. 2018;17(9):641-59.
24. Liao F, Li A, Xiong M, Bien-Ly N, Jiang H, Zhang Y, et al. Targeting of nonlipidated, aggregated apoE with antibodies inhibits amyloid accumulation. The Journal of clinical investigation. 2018;128(5):2144-55.
25. Choudhury SR, Harris AF, Cabral DJ, Keeler AM, Sapp E, Ferreira JS, et al. Widespread central nervous system gene transfer and silencing after systemic delivery of novel AAV-AS vector. Molecular Therapy. 2016;24(4):726-35.
26. Ravina B, Christine C, Bankiewicz K, Van Laar A, Richardson M, Kells A, et al., editors. Intraputaminal AADC gene therapy for advanced Parkinson's disease: interim results of a phase 1b Trial. Human Gene Therapy; 2017: MARY ANN LIEBERT, INC 140 HUGUENOT STREET, 3RD FL, NEW ROCHELLE, NY 10801 USA.
27. Leone P, Shera D, McPhee SW, Francis JS, Kolodny EH, Bilaniuk LT, et al. Long-term follow-up after gene therapy for canavan disease. Science translational medicine. 2012;4(165):165ra3-ra3.
28. Jamalidoust M, Ravanshad M, Namayandeh M, Zare M, Asaei S, Ziyaeyan M. Construction of AAV-rat-IL4 and Evaluation of its Modulating Effect on Aβ (1-42)-Induced Proinflammatory Cytokines in Primary Microglia and the B92 Cell Line by Quantitative PCR Assay. Jundishapur journal of microbiology. 2016;9(3):e30444-e.
29. VandenDriessche T, Chuah MK. Hemophilia gene therapy: ready for prime time? Human gene therapy. 2017;28(11):1013-23.
30. Spencer H, Riley B, Doering C. State of the art: gene therapy of haemophilia. Haemophilia. 2016;22:66-71.
31. Batty P, Lillicrap D. Hemophilia Gene Therapy: Approaching the First Licensed Product. HemaSphere. 2021;5(3).
32. Dodd M, Marquez‐Curtis L, Janowska‐Wieczorek A, Hortelano G. Sustained expression of coagulation factor IX by modified cord blood‐derived mesenchymal stromal cells. The journal of gene medicine. 2014;16(5-6):131-42.
33. Bulcha JT, Wang Y, Ma H, Tai PWL, Gao G. Viral vector platforms within the gene therapy landscape. Signal Transduction and Targeted Therapy. 2021;6(1):53.
34. Martinez-Lage M, Puig-Serra P, Menendez P, Torres-Ruiz R, Rodriguez-Perales S. CRISPR/Cas9 for Cancer Therapy: Hopes and Challenges. Biomedicines. 2018;6(4):105.
35. Yin H, Song C-Q, Dorkin JR, Zhu LJ, Li Y, Wu Q, et al. Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo. Nature biotechnology. 2016;34(3):328-33.
36. Yin H, Kauffman KJ, Anderson DG. Delivery technologies for genome editing. Nature reviews Drug discovery. 2017;16(6):387.
37. Gao J, Bergmann T, Zhang W, Schiwon M, Ehrke-Schulz E, Ehrhardt A. Viral vector-based delivery of CRISPR/Cas9 and donor DNA for homology-directed repair in an in vitro model for canine hemophilia B. Molecular Therapy-Nucleic Acids. 2019;14:364-76.
38. Ates I, Rathbone T, Stuart C, Bridges PH, Cottle RN. Delivery approaches for therapeutic genome editing and challenges. Genes. 2020;11(10):1113.
39. Warnock JN, Daigre C, Al-Rubeai M. Introduction to viral vectors. Viral Vectors for Gene Therapy: Springer; 2011. p. 1-25.