Release Date: 2024-02-12

Biological Methods Used in Gene Therapy

Baris Gulhan (Author)

Release Date: 2024-02-12

Gene therapy is used to prevent or treat genetic diseases. To treat genetic diseases, transfer of genes that correct the effects of the mutation that causes the disease to the patient. In gene therapy, the therapeutic gene is transferred to target cells through a vector and viral vectors are most used. Retroviruses (lentiviruses), adenoviruses, adeno-associated [...]

Media Type
    Buy from

    Price may vary by retailers

    Work TypeBook Chapter
    Published inGene Therapy
    First Page39
    Last Page63
    DOIhttps://doi.org/10.69860/nobel.9786053358824.3
    Page Count25
    Copyright HolderNobel Tıp Kitabevleri
    Licensehttps://nobelpub.com/publish-with-us/copyright-and-licensing
    Gene therapy is used to prevent or treat genetic diseases. To treat genetic diseases, transfer of genes that correct the effects of the mutation that causes the disease to the patient. In gene therapy, the therapeutic gene is transferred to target cells through a vector and viral vectors are most used. Retroviruses (lentiviruses), adenoviruses, adeno-associated viruses, and herpes virus vectors are common as viral vectors. In this section, biological methods used especially in gene therapy stages examined.

    Baris Gulhan (Author)
    Associate Professor, Erzincan Binali Yıldırım University
    https://orcid.org/0000-0002-2605-1282
    3Associate Prof. Dr. Barış GÜLHAN is a scientist with a professional career spanning over twenty years and original contributions in the field of medical microbiology. He was born in Gölcük/İzmit, Turkiye. He completed his undergraduate education in 2000 in the Erciyes University Medical Faculty. He worked as a practitioner doctor for about 4 years. In 2005, he started his specialty education at Dicle University and completed it in 2009 and he became a specialist in the medical microbiology field. In July 2012 he began as an assistant professor in the medical microbiology department of Erzincan Binali Yıldırım University Medical Faculty. In August 2023 he has an associate professor degree. His work has led to the publication of more than 72 research articles, oral and poster presentations, as well as 8 research projects. He is currently working at Erzincan Binali Yıldırım University Medical Faculty, in the Department of Medical Microbiology.

    • Rashnonejad A, Durmaz B, Özkınay F. The principles of gene therapy and recent advances. Ege Journal of Medicine 2014;53(4):231-240

    • Akhtar N, Akram M, Asif HM, Usmanghani K, Shah SMA, Rao SA, Uzair M, Shaheen G, Ahmad K. Gene therapy: A review article. Journal of Medicinal Plants Research 2011; 5(10): 1812-1817

    • Haritha PN, Devi SKU, Nagaratna DP, Chaitanya PSK, Gunasekharan V. Gene therapy- a review. Int J Biopharmaceutics 2012;3(1):55-64

    • Naldini L. Gene therapy returns to centre stage. Nature. 2015; 526: 351-360.

    • Hasan N, Saini S. Gene Therapy: Current status and future perspective. International Journal of Pharma Sciences and Research 2014;5: 586-593.

    • Nayerossadat N, Maedeh T, Ali PA. Viral and nonviral delivery systems for gene delivery. Advanced Biomedical Research 2012; 1(2): 1-27.

    • Soofiyani S, Baradaran B, Lotfipour F, Kazemi T, Mohammadnejad L.Gene Therapy, Early Promises, Subsequent Problems, and Recent Breakthroughs. Advanced Pharmaceutical Bulletin 2013; 3(2), 249-255.

    • Smith KR. Gene therapy: The potential applicability of gene transfer technology to the human germline. Int J Med Sci 2004;1(2):76-91

    • Nielsen TO. Human germline gene therapy. MJM 1997;3(2):126-32.

    • Suhonen J, Ray J, Blömer U, Gage FH, Kaspar B. Ex vivo and in vivo gene delivery to the brain. Curr Protoc Hum Genet. 2006; 51: 1-25.

    • Gregory-Evans K, Bashar AM, Tan M. Ex vivo gene therapy and vision. Curr Gene Ther. 2012; 12(2):103-15.

    • Castellani S, Conese M. Lentiviral Vectors and Cystic Fibrosis Gene Therapy. Viruses. 2010; 2: 395-412.

    • Matraı J, Chuah MK, Vandendrıessche T. Recent advances in lentiviral vector development and applications. Mol. Ther. 2010; 18, 477-490.

    • Adamson CS, Jones IM. The molecular basis of HIV capsid assemblyfive years of progress. Reviews in medical virology. 2012; 14(2):107–21.

    • Bukrınsky MI, Haffar OK. HIV-1 nuclear import: in search of a leader. Front. Biosci. 1999; 4, 772-781.

    • Freed E.O. HIV-1 replication. Somat. Cell Mol. Genet. 2001; 26: 13-33.

    • Katz RA, Skalka AM. The retroviral enzymes. Annual review of biochemistry. 1994; 63:133–73.

    • Pluta K, Kacprzak MM. Use of HIV as a gene transfer vector. Acta biochimica Polonica. 2009; 56(4):531–95.

    • Dropulı B. Lentiviral vectors: their molecular design, safety, and use in laboratory and preclinical research. Hum. Gene Ther. 2011; 22: 649-657.

    • Howarth JL, Youn Bok Lee Y, Uney JB. Using viral vectors as gene transfer tools (Cell Biology and Toxicology Special Issue: ETCS-UK 1 day meeting. Cell. Biol. Toxicol. 2010; 6: 1-20.

    • Vannucci L, Lai M, Chiuppesi F, Ceccherini-Nelli L, Pistello M. Viral vectors: a look back and ahead on gene transfer technology. New Microbiologica, 2013; 36(1):1-22.

    • Yang, Z. R. et al. Recent developments in the use of adenoviruses and immunotoxins in cancer gene therapy. Cancer Gene Ther. 2007; 14: 599–615.

    • Russell, W. C. Adenoviruses: update on structure and function. J. Gen. Virol. 2009; 90: 1–20.

    • Saban, S. D., Silvestry, M., Nemerow, G. R. & Stewart, P. L. Visualization of alphahelices in a 6-angstrom resolution cryoelectron microscopy structure of adenovirüs allows refinement of capsid protein assignments. J. Virol. 2006; 80: 12049–12059.

    • San Martin, C. et al. Localization of the N-terminus of minor coat protein IIIa in the adenovirus capsid. J. Mol. Biol. 383, 923–934 (2008).

    • Fabry, C. M. et al. A quasi-atomic model of human adenovirus type 5 capsid. EMBO J. 2005; 24: 1645–1654.

    • Ahi, Y. S. & Mittal, S. K. Components of adenovirus genome packaging. Front Microbiol. 2016; 7: 1503.

    • Tomko, R. P., Xu, R. & Philipson, L. HCAR and MCAR: the human and mouse cellular receptors for subgroup C adenoviruses and group B coxsackieviruses. Proc. Natl Acad. Sci. Usa. 1997; 94, 3352–3356.

    • Bergelson, J. M. et al. Isolation of a common receptor for Coxsackie B viruses and adenoviruses 2 and 5. Science 1997; 275: 1320–1323.

    • Gaden, F. et al. Gene transduction and cell entry pathway of fiber-modified adenovirus type 5 vectors carrying novel endocytic peptide ligands selected on human tracheal glandular cells. J. Virol. 2004; 78: 7227–7247.

    • Wiethoff, C. M. & Nemerow, G. R. Adenovirus membrane penetration: tickling the tail of a sleeping dragon. Virology 2015; 479-480: 591–599.

    • Bremner, K. H. et al. Adenovirus transport via direct interaction of cytoplasmic dynein with the viral capsid hexon subunit. Cell Host Microbe 2009;6: 523–535.

    • Lee, C. S. et al. Adenovirus-mediated gene delivery: potential applications for gene and cell-based therapies in the new era of personalized medicine. Genes Dis. 2017; 4: 43–63.

    • Kotin, R. M. et al. Site-specific integration by adeno-associated virus. Proc. Natl Acad. Sci. USA, 1990; 87: 2211–2215.

    • Samulski, R. J., Berns, K. I., Tan, M. & Muzyczka, N. Cloning of adeno-associated virus into pBR322: rescue of intact virus from the recombinant plasmid in human cells. Proc. Natl Acad. Sci. US, 1982; 79: 2077–2081.

    • Calcedo, R. et al. Worldwide epidemiology of neutralizing antibodies to adenoassociated viruses. J. Infect. Dis. 2009; 199: 381–390.

    • Flotte, T. et al. A phase I study of an adeno-associated virus-CFTR gene vector in adult CF patients with mild lung disease. Hum. Gene Ther. 1996; 7: 1145–1159.

    • Nakai, H., Storm, T. A. & Kay, M. A. Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors. Nat. Biotechnol. 2000; 18: 527–532.

    • Sun, L., Li, J. & Xiao, X. Overcoming adeno-associated virus vector size limitation through viral DNA heterodimerization. Nat. Med. 2000; 6: 599–602.

    • Ghosh, A., Yue, Y., Lai, Y. & Duan, D. A hybrid vector system expands adenoassociated viral vector packaging capacity in a transgene-independent manner. Mol. Ther. 2008; 16: 124–130.

    • Flotte, T. et al. A phase I study of an adeno-associated virus-CFTR gene vector in adult CF patients with mild lung disease. Hum. Gene Ther. 1996; 7: 1145–1159.

    • Duan, D., Yue, Y., Yan, Z. & Engelhardt, J. F. A new dual-vector approach to enhance recombinant adeno-associated virus-mediated gene expression through intermolecular cis activation. Nat. Med. 2000; 6: 595–598.

    • Roızman B., Knıpe D., Whıtley R. Herpes simplex viruses, p. 25012602. In: H.P. Knipe DM, ed. Fields Virology, 5th ed ed. Lippincott Williams & Wilkins, Philadelphia, 2007.

    • Chou J, Kern ER, Whitley RJ, et al. Mapping of herpes simplex virus-1 neurovirulence to γ134.5, a gene nonessential for growth in culture. Science. 1990; 250: 1262- 1266.

    Share This Chapter!