TREATMENT POSSIBILITIES FOR ACQUIRED AND HEREDITARY DISEASES BY GENE THERAPY: A REVIEW
Therapeutic nucleic acids demand specificity and accuracy in design as well as delivery strategies used in replacement or silencing of the target gene. Gene therapy is believed to be the therapy in which the root cause of the diseases can be treated at the molecular level. Generally gene therapy helps in the identification of the origin of the disorder instead of using drugs to diminish or control the symptoms. The application of nucleic acids to treat and control diseases is known as “gene therapy.” Gene therapy consists on the substitution or addition of a functional gene into the nucleus of a living cell, in order to treat a disease or repair a dysfunction, caused by this gene failure. This therapy is used to correct defective genes, which are responsible for genetic diseases. Thus, gene therapy can be used to prevent, treat or regulate hereditary or acquired disorders, by the production of therapeutic proteins. The gene therapy is mediated by the use of viral and non-viral vectors to transport foreign genes into somatic cells to restorative defective genes. This review focuses on viral vectors in detail.
2. Zhu L, Mahato RI. Lipid and polymeric carrier-mediated nucleic acid delivery. Expert Opin Drug Delivery 2010;7:1209-26.
3. Wolff JA, Malone RW, Williams P, Chong W, Acsadi G, Jani A. Direct gene transfer into mouse muscle in vivo. Science 1990;247:1465-8.
4. Gill HS, Prausnitz MR. Coated microneedles for transdermal delivery. J Controlled Release 2007;117:227-37.
5. Fire A, Xu S, Montgomery MK, Kostas SA, Driver SE, Mello CC. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature 1998;391:806-11.
6. Patil PM, Chaudhari PD, Megha S, Duragkar NJ. Review article on gene therapy. Int J Genetics 2012;4:74-9.
7. Al-Lawatia YMM, Mullaicharam AR. Viral vectors for gene therapy: a review. Res J Pharm Biol Chem Sci 2013;4:813-23.
8. Cevher E, Sezer AD, Calar E. In: Gene delivery systems: Recent progress in viral and non-viral therapy. Sezer AD. Ed. In tech: Croatia; 2012. p. 337-470.
9. Pathak A, Patnaik S, Gupta KC. Recent trends in non-viral vector-mediated gene delivery. Biotechnol J 2009;4:1559-72.
10. Yin H, Kanasty RL, Eltoukhy AA, Vegas AJ, Dorkin JR, Anderson DG. Non-viral vectors forgene-based therapy. Nat Rev Gene 2014;15:54155.
11. Harada Shiba M, Yamauchi K, Harada A, Takamisawa I, Shimokado K, Kataoka K. Polyioncomplex micelles as vectors in gene therapy-pharmacokinetics and in vivo gene transfer. Gene Ther 2002;9:407-14.
12. Kariko K, Bhuyan P, Capodici J, Weissman D. Small interfering RNAs mediate sequence-independent gene suppression and induce immune activation by signaling through tolllike receptor 3. J Immunol 2004;172:6545-9.
13. Yoshida H, Nishikawa M, Yasuda S, Mizuno Y, Toyota H, Kiyota T, et al. TLR9-dependent systemic interferon-beta production by intravenous injection of plasmid DNA/cationic liposome complex in mice. J Gene Med 2009;11:708-17.
14. Bieber T, Meissner W, Kostin S, Niemann A, Elsasser HP. Intracellular route and transcriptional competence of polyethyleneimine-DNA complexes. J Controlled Release 2002;82:441-54.
15. Liu G, Li D, Pasumarthy MK, Kowalczyk TH, Gedeon CR, Hyatt SL, et al. Nanoparticles of compacted DNA transfect post-mitotic cells. J Biol Chem 2003;278:32578-86.
16. Roth CM, Sundaram S. Engineering synthetic vectors for improved DNA delivery: insights from intracellular pathways. Annu Rev Biomed Eng 2004;6:397-426.
17. Zhu L, Lu Y, Miller DD, Mahato RI. Structural and formulation factors are influencing pyridinium lipid-based gene transfer. Bioconjugate Chem 2008;19:2499-512.
18. Belting M, Sandgren S, Wittrup A. Nuclear delivery of macromolecules: barriers and carriers. Adv Drug Delivery Rev 2005;57:505-27.
19. Read ML, Logan A, Seymour LW. Barriers to gene delivery using synthetic vectors. Adv Genet 2005;53:19-46.
20. Luo D, Saltzman WM. Synthetic DNA delivery systems. Nat Biotech 2000a;18:33-7.
21. Yin W, Xiang P, Li Q. Investigations of the effect of DNA size in transient transfection assay using dual luciferase system. Anal Biochem 2005;346:289-94.
22. Williams JA, Carnes AE, Hodgson CP. Plasmid DNA vaccine vector design impact on efficacy, safety and upstream production. Biotechnol Adv 2009;27:353-70.
23. Asoh S, Lee Kwon W, Mouradian MM, Nirenberg M. Selection of DNA clones with enhancer sequences. Proc Natl Acad Sci 1994;9:6982-6.
24. Noss KR, Wolfe SA, Grimes SR. Upregulation of prostate-specific membrane antigen/folatehydrolase transcription by an enhancer. Gene 2002;285:247-56.
25. Williford MJ, Wu J, Ren Y, Archang MM, Leong KW, Mao HQ. Recent advances innanoparticle-mediatedsiRNA delivery. Annu Rev Biomed Eng 2014;16:347-70.
26. Bumcrot D, Manoharan M, Koteliansky V, Sah DWY. RNAi therapeutics: a potential new class of pharmaceutical drugs. Nat Chem Biol 2006;2:711-9.
27. Agrawal N, Dasaradhi PVN, Mohmmed A, Malhotra P, Bhatnagar RK, Mukherjee SK. RNA interference: biology, mechanism, and applications. Microbiol Mol Biol Rev 2003;67:657-85.
28. Fougerolles A, Vornlocher HP, Maraganore J, Lieberman J. Interfering with disease: a progressreport on siRNA-based therapeutics. Nat Rev Drug Discovery 2007;6:443-53.
29. Grimm D, Kay MA. Therapeutic application of RNAi: is mRNA targeting finally ready forprime time? J Clin Invest 2007;117:3633-41.
30. Preall JB, Sontheimer EJ. RNAi: RISC gets loaded. Cell 2005;123:543-5.
31. Hammond SM, Boettcher S, Caudy AA, Kobayashi R, Hannon GJ. Argonaute2, a link between genetic and biochemical analyses of RNAi. Science 2001;93:1146-50.
32. Watts JK, Deleavey GF, Damha MJ. Chemically modified siRNA: tools and applications. Drug Discovery Today 2008;13:842-55.
33. Paddison PJ, Caudy AA, Bernstein E, Hannon GJ, Conklin DS. Short hairpin RNAs (shRNAs)induce sequence-specific silencing in mammalian cells. Genes Dev 2002;16:948-58.
34. Chen Y, Cheng G, Mahato RI. RNAi for treating hepatitis B viral infection. Pharm Res 2008;25:72-86.
35. Rao DD, Vorhies JS, Senzer N, Nemunaitis J. siRNA vs. shRNA: similarities and differences. Adv Drug Delivery Rev 2009;61:746-59.
36. Lee Y, Jeon K, Lee JT, Kim S, Kim VN. MicroRNA maturation: stepwise processing and subcellular localization. EMBO J 2002;21:4663-70.
37. Zeng Y, Cullen BR. Structural requirements for pre-microRNA binding and nuclear export by exportin 5. Nucleic Acids Res 2004;32:4776-85.
38. Yang M, Mattes J. Discovery, biology and therapeutic potential of RNA interference, micro-RNA and antagomirs. Pharmacol Ther 2008;117:94-104.
39. Phillips JE, Gersbach CA, Garcia AJ. Virus-based gene therapy strategies for bone regeneration. Biomaterials. 2007;28:211-29.
40. Huang S, Kamihira M. Development of hybrid viral vectors for gene therapy. Biotechnol Adv 2013;31:208-23.
41. Seth P. Vector-mediated cancer gene therapy: an overview. Cancer Biol Ther 2005;4:512-7.
42. Cevher E, Sezer AD, Caglar E. Gene delivery systems: recent progress in viral and non-viral therapy. Recent Adv Novel Drug Carrier Syst 2012:437-70. DOI:10.5772/53392
43. Akhtar N, Akram M, Asif HM, Usmanghani K, Shah SA, Rao SA, et al. Gene therapy: a review article. J Med Plants Res 2011;5:1812-7.
44. Walther W, Stein U. Viral vectors for gene transfer. Drugs 2000;60:249-71.
45. Mahato RI, Smith LC, Rolland A. Pharmaceutical perspectives of nonviral gene therapy. In: Advances in genetics. Academic Press 1999;41:95-156.
46. Levesque C. Vector-mediated gene therapy and the herpes simplex virus. BioTeach J 2004;2:112-6.
47. Kay MA, Glorioso JC, Naldini L. Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics. Nat Med 2001;7:33-40.
48. Lundstrom K, Boulikas T. Viral and non-viral vectors in gene therapy: technology development and clinical trials. Technol Cancer Res Treat 2003;2:471-85.
49. Daya S, Berns KI. Gene therapy using adeno-associated virus vectors. Clin Microbiol Rev 2008;21:583-93.
50. Misra S. Human gene therapy: a brief overview of the genetic revolution. J Assoc Physicians India 2013;61:127-33.
51. Gad SC. editor. Handbook of pharmaceutical biotechnology. John Wiley and Sons; 2007.
52. Thomas CE, Ehrhardt A, Kay MA. Progress and problems with the use of viral vectors for gene therapy. Nat Rev Genetics 2003;4:346-58.
53. Crommelin DJ. Formulation of biotech products, including biopharmaceutical considerations. In: Pharmaceutical biotechnology. Springer, New York NY; 2013. p. 69-99.
54. Bleiziffer O, Eriksson E, Yao F, Horch RE, Kneser U. Gene transfer strategies in tissue engineering. J Cell Mol Med 2007;11:206-23.
55. Stone D, David A, Bolognani F, Lowenstein PR, Castro MG. Viral vectors for gene delivery and gene therapy within the endocrine system. J Endocrinol 2000;164:103-18.
56. Maurya SK, Srivastava S, Joshi RK. Retroviral vectors and gene therapy: an update. Indian J Biotechnol 2009;8:349-57.
57. Vigna E, Naldini L. Lentiviral vectors: excellent tools for experimental gene transfer and promising candidates for gene therapy. J Gene Med 2000;2:308-16.
58. Escors D, Breckpot K. Lentiviral vectors in gene therapy: their current status and future potential. Arch Immunol Ther Exp 2010;58:107-19.
59. Cockrell AS, Kafri T. Gene delivery by lentivirus vectors. Mol Biotechnol 2007;36:184-204.
60. C Silva A, M Lopes C, M Sousa Lobo J, Helena Amaral M. Nucleic acids delivery systems: a challenge for pharmaceutical technologists. Curr Drug Metab 2015;16:3-16.
61. Volpers C, Kochanek S. Adenoviral vectors for gene transfer and therapy. J Gene Med 2004;6(S1):S164-71.
62. Gill DR, Davies LA, Pringle IA, Hyde SC. The development of gene therapy for diseases of the lung. Cell Mol Life Sci 2004;61:355-68.
63. Kootstra NA, Verma IM. Gene therapy with viral vectors. Annu Rev Pharmacol Toxicol 2003;43:413-39.
64. Manservigi R, Argnani R, Marconi P. HSV recombinant vectors for gene therapy. Open Virol J 2010;4:123.
65. Vannucci L, Lai M, Chiuppesi F, Ceccherini-Nelli L, Pistello M. Viral vectors: a look back and ahead on gene transfer technology. New Microbiol 2013;36:1-22.
66. Kanesa Thasan N, Smucny JJ, HokeJr CH, Marks DH, Konishi E, Kurane I, et al. Safety and immunogenicity of NYVAC-JEV and ALVAC-JEV attenuated recombinant Japanese encephalitis virus—poxvirus vaccines in vaccinia-nonimmune and vaccinia-immune humans. Vaccine 2000;19:483-91.
67. Doehn C, Kausch I, Boehmer T, Sommerauer M, Jocham D. Drug evaluation: Therion'srV-PSA-TRICOM+rF-PSA-TRICOM prime-boost prostate cancer vaccine. Curr Opin Mol Ther 2007;9:183.
68. Kirn DH, Thorne SH. Targeted and armed oncolytic poxviruses: a novel multi-mechanistic therapeutic class for cancer. Nat Rev Cancer 2009;9:64-71.
69. Lam PY, Breakefield XO. Hybrid vector designs to control the delivery, fate and expression of transgenes. J Gene Med 2000;2:395-408.
70. Gonçalves MA. Adeno-associated virus: from defective virus to effective vector. Virol J 2005;2:43.
71. Reynolds PN, Feng M, Curiel DT. Chimeric viral vectors the best of both worlds? Mol Med Today 1999;5:25-31.
72. Fraefel C, Jacoby DR, Lage C, Hilderbrand H, Chou JY, Alt FW, Breakefield XO, et al. Gene transfer into hepatocytes mediated by helper virus-free HSV/AAV hybrid vectors. Mol Med 1997;3:813-25.
73. Costantini LC, Wang S, Fraefel C, Breakefield XO, Isacson O. Gene transfer to the nigrostriatal system by hybrid herpes simplex virus/adeno-associated virus amplicon vectors. Human Gene Ther 1999;10:2481-94.
74. Hildinger M, Auricchio A, Gao G, Wang L, Chirmule N, Wilson JM. Hybrid vectors based on adeno-associated virus serotypes 2 and 5 for muscle-directed gene transfer. J Virol 2001;75:6199-203.
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