Gene therapy is a new area in medicine

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The review gives information on the history and areas of gene therapy development. It discusses the types of transgenes used to treat different diseases; specific and constitutional promoters that ensure the expression of therapeutic transgenes in targeted cells; procedures for delivering gene constructions into the targeted cells; and mechanisms by which the genetic material is introduced into the cell nucleus. The advantages and disadvantages of various types of viral and nonviral vectors are considered in detail. There is recent evidence on the clinical application of officinal drugs for gene therapy, which are registered for the treatment of patients with head and neck cancer (Gendicine and Oncorine, China), the monogenic disease lipoprotein lipase deficiency (Glybera, the Netherlands), or atherosclerotic lower extremity ischemia (Neovaskulgen, Russia), and on clinical tests of medications for gene therapy in different fields of medicine.

Keywords:

gene therapy

transgene

promoter

vector

Authors:

Bezborodova O.A.

P.A. Herzen Moscow Oncology Research Institute, Branch, National Medical Radiology Research Center, Ministry of Health of the Russian Federation, Moscow, Russia

Nemtsova E.R.

P.A. Herzen Moscow Oncology Research Institute, Branch, National Medical Radiology Research Center, Ministry of Health of the Russian Federation, Moscow, Russia

Iakubovskaia R.I.

FGBU "Moskovskiĭ nauchno-issledovatel'skiĭ onkologicheskiĭ institut im. P.A. Gertsena" Minzdravsotsrazvitiia Rossii

Kaprin A.D.

FGBU "Moskovskiĭ nauchno-issledovatel'skiĭ onkologicheskiĭ institut im. P.A. Gertsena" Minzdrava Rossii

SPIN RSCI: 1759-8101
Scopus AuthorID: 6602709853
ResearcherID: K-1445-2014
ORCID: 0000-0001-8784-8415

List of references:

Available at: http://www.wiley.co.uk/genmed/clinical
Wang D, Zhong L, Nahid MA, Gao G. The potential of adeno-associated viral vectors for gene delivery to muscle tissue. Expert Opin Drug Deliv. 2014;11(3):345-364. doi:10.1517/17425247.2014.871258.
Jazwa A, Florczyk U, Jozkowicz A, Dulak J. Gene therapy on demand: site specific regulation of gene therapy. Gene. 2013;525(2):229-238. doi:10.1016/j.gene.2013.03.093.
Akyurek LM, Yang ZY, Aoki K, San H, Nabel GJ, Parmacek MS et al. SM22alpha promoter targets gene expression to vascular smooth muscle cells in vitro and in vivo. Mol Med. 2000;6(11):983-991.
Lee SJ, Kim HS, Yu R, Bae KH, Stantz KM, Lee SJ et al. Novel prostate-specific promoter derived from PSA and PSMA enhancers. Mol Ther. 2002;6(3):415-421.
Smith MJ, Rousculp MD, Goldsmith KT, Curiel DT, Garver RI. Surfactant protein A-directed toxin gene kills lung cancer cells in vitro. Hum Gene Ther. 1994;5(1):29-35.
Strayer MS, Guttentag SH, Ballard PL. Targeting type II and Clara cells for adenovirus-mediated gene transfer using the surfactant protein B promoter. Am J Respir Cell Mol Biol. 1998;18(1):1-11.
Moehle C, Ackermann N, Langmann T, Aslanidis C, Kel A, Kel-Margoulis O et al. Aberrant intestinal expression and allelic variants of mucin genes associated with inflammatory bowel disease. J Mol Med. 2006;84(12):1055-1066.
Kuriyama S, Yoshikawa М, Ishizaka S, Tsujli T, Ikenaka K, Kagawa T et al. A potential approach for gene therapy targeting hepatoma using a liver-specific promoter on a retroviral vector. Cell Struct Funct. 1991;16(6):503-510.
Van Houdt WJ, Haviv YS, Lu B, Wang M, Rivera AA, Ulasov IV et al. The human survivin promoter: a novel transcriptional targeting strategy for treatment of glioma. J Neurosurg. 2006;104(4):583-592.
Gu J, Fang B. Telomerase promoter-driven cancer gene therapy. Cancer Biol Ther. 2003;2(4,Suppl.1):S64-S70.
Thomas SM, Grandis JR. The current state of head and neck cancer gene therapy. Hum Gene Ther. 2009;20(12):1565-1575. doi:10.1089/hum.2009.163.
Wang W, Li W, Ma N, Steinhoff G. Non-viral gene delivery methods. Curr Pharm Biotechnol. 2013;14(1):46-60.
Thomas CE, Ehrhardt A, Kay MA. Progress and problems with the use of viral vectors for gene therapy. Nat Rev Genet. 2003;4(5):346-358.
Yuan Z, Pastoriza J, Quinn T, Libutti S. Targeting tumor vasculature using adeno-associated virus page vector coding tumor necrosis factor-a. In: Lattime EC, Gerson SL, eds. Gene therapy of cancer. 3rd ed. San Diego (CA): Elsevier; 2013:19-33. doi:10.1038/cgt.2012.83.
Hacein-Bey-Abina S, Hauer J, Lim A, Picard C, Wang GP, Berry CC et al. Efficacy of gene therapy for X-linked severe combined immunodeficiency. N Engl J Med. 2010;363(4):355-364. doi:10.1056/NEJMoa1000164.
Bangari DS, Mittal SK. Current strategies and future directions for eluding adenoviral vector immunity. Curr Gene Ther. 2006;6(2):215-226.
Palmer DJ, Ng P. Helper-dependent adenoviral vectors for gene therapy. Hum Gene Ther. 2005;16(1):1-16.
Wen S, Graf S, Massey PG, Dichek DA. Improved vascular gene transfer with a helper-dependent adenoviral vector. Circulation. 2004;110(11):1484-1491.
Jin L, Zeng X, Liu M, Deng Y, He N. Current progress in gene delivery technology based on chemical methods and nano-carriers. Theranostics. 2014;4(3):240-255. doi:10.7150/thno.6914.
Fang B, Roth JA. Tumor-suppressing gene therapy. Cancer Biol Ther. 2003;2(4,Suppl.1):S115-S121.
Roth JA. Adenovirus p53 gene therapy. Expert Opin Biol Ther. 2006;6:55-61.
Peng Z. Current status of gendicine in China: recombinant Ad-p53 agent for treatment of cancers. Hum Gene Ther. 2005;16:1016-1027.
Fung H, Gersson S. Viral insertion site detection and analysis in cancer gene therapy. In: Lattime EC, Gerson SL, eds. Gene therapy of cancer. 3rd ed. San Diego (CA): Elsevier; 2013:35-46.
Gujrati M, Lu Z. Targeted systemic delivery of therapeutic siRNA. In: Lattime EC, Gerson SL, eds. Gene therapy of cancer. 3rd ed. San Diego (CA): Elsevier; 2013:47-65.
Davis ME. The first targeted delivery of siRNA in humans via a self-assembling, cyclodextrin polymer-based nanoparticle: from concept to clinic. Mol Pharm. 2009;6(3):659-668. doi:10.1021/mp900015y.
Tabernero J, Shapiro GI, LoRusso PM, Cervantes A, Schwartz GK, Weiss GJ et al. First-in-humans trial of an RNA interference therapeutic targeting VEGF and KSP in cancer patients with liver involvement. Cancer Discov. 2013;3:406-417. doi:10.1158/2159-8290.CD-12-0429.
Freeman SM, Whartenby KA, Freeman JL, Abboud CN, Marrogi AJ. In situ use of suicide genes for cancer therapy. Semin Oncol. 1996;23:31-45.
Duarte S, Carle G, Faneca H, de Lima MC, Pierrefite-Carle V. Suicide gene therapy in cancer: Where do we stand now? Cancer Lett. 2012;324(2):160-170. doi:10.1016/j.canlet.2012.05.023.
Immonen A, Vapalahti M, Tyynela K, Hurskainen H, Sandmair A, Vanninen R et al. AdvHSV-tk gene therapy with intravenous ganciclovir improves survival in human malignant glioma: a randomized, controlled study. Mol Ther. 2004;10(5):967-972.
van Putten EH, Dirven CM, van den Bent MJ, Lamfers ML. Sitimagene ceradenovec: a gene-based drug for the treatment of operable high-grade glioma. Future Oncol. 2010;6(11):1691-1610. doi:10.2217/fon.10.134.
Aghi M, Martuza RL. Oncolytic viral therapies — the clinical expirience. Oncogene. 2005;24:7802-7816.
Heise C, Ganly I, Kim YT, Sampson-Johannes A, Brown R, Kirn D. Efficacy of a replication-selective adenovirus against ovarian carcinomatosis is dependent on tumor burden, viral replication and p53 status. Gene Ther. 2000;7:1925-1929.
Available at: http://www.oncolyticsbiotech.com/clinical.html
Hu JC, Coffin RS, Davis CJ, Graham NJ, Groves N, Guest PJ et al. A phase I study of OncoVEXGM-CSF, a second-generation oncolytic herpes simplex virus expressing granulocyte macrophage colony-stimulating factor. Clin Cancer Res. 2006;12(22):6737-6747.
Hodge JW, Sabzevari H, Yafal AG, Gritz L, Lorenz MG, Schlom J. A triad of costimulatory molecules synergize to amplify T-cell activation. Cancer Res. 1999;59:5800-5807.
Aarts WM, Schlom J, Hodge JW. Vector-based vaccine/cytokine combination therapy to enhance induction of immune responses to a self-antigen and antitumor activity. Cancer Res. 2002;62:5770-5777.
Hodge JW, Poole DJ, Aarts WM, Gomez Yafal A, Gritz L, Schlom J. Modified vaccinia virus ankara recombinants are as potent as vaccinia recombinants in diversified prime and boost vaccine regimens to elicit therapeutic antitumor responses. Cancer Res. 2003;63:7942-7949.
Kantoff PW, Schuetz TJ, Blumenstein BA, Glode LM, Bilhartz DL, Wyand M et al. Overall survival analysis of a phase II randomized controlled trial of a Poxviral-based PSA-targeted immunotherapy in metastatic castration-resistant prostate cancer. J Clin Oncol. 2010;28:1099-1105. doi:10.1200/JCO.2009.25.0597.
Heery CR, Madan RA, Bilusic M, Kim JW, Singh NK. Interim analysis of a phase 2 randomized clinical trial of samarium-153 (Sm-153) with or without PSA-TRICOM vaccine in metastatic castration-resistant prostate cancer after docetaxel. J Clin Oncol. 2012;30(15,Suppl.):abstr.2526. (ASCO Annual Meeting. June 1-5, Chicago; 2012.)
Docetaxel alone or in combination with vaccine to treat breast cancer. Docetaxel and prednisone with or without vaccine therapy in treating patients with metastatic hormone-resistant prostate cancer. Available at: http://www.clinicaltrials.gov/
Turtle CJ. Chimeric antigen receptor modified T cell therapy for B cell malignancies. Int J Hematol. 2014;99(2):132-140. doi:10.1007/s12185-013-1490-x.
Xu Y, Zhang M, Ramos CA, Durett A, Liu E, Dakhova O et al. Closely related T-memory stem cells correlate with in vivo expansion of CAR.CD19-T cells and are preserved by IL-7 and IL-15. Blood. 2014;123(24):3750-3759. doi:10.1182/blood-2014-01-552174.
Kochenderfer JN, Dudley ME, Feldman SA, Wilson WH, Spaner DE, Maric I et al. B-cell depletion and remissions of malignancy along with cytokine-associated toxicity in a clinical trial of anti-CD19 chimeric-antigen-receptor-transduced T cells. Blood. 2012;119(12):2709-2720. doi:10.1182/blood-2011-10-384388.
Marcus A, Eshhar Z. Allogeneic chimeric antigen receptor-modified cells for adoptive cell therapy of cancer. Expert Opin Biol Ther. 2014;14(7):947-954. doi:10.1517/14712598.2014.900540.
Ma G, Shimada H, Hiroshima K, Tada Y, Suzuki N, Tagawa M. Gene medicine for cancer treatment: Commercially available medicine and accumulated clinical data in China Dev Ther. 2008;2:115-122.
Wierzbicki AS, Viljoen A. Alipogene tiparvovec: gene therapy for lipoprotein lipase deficiency. Expert Opin Biol Ther. 2013;13(1):7-10. doi:10.1517/14712598.2013.738663.
Deev R. Gene therapy in Russia: three years of experience. Khimiya i zhizn'-XXI vek. 2013;12. Available at: http://www.hij.ru (In Russ.)
Available et: http://www.dissercat.com/content/strategiya-sozdaniya-tselnokletochnykh-antimelanomnykh-vaktsin. (In Russ.)
Vdovichenko G, Petrishchenko V, Sergeev A, Kim I, Fatyukhina O, Shishkina L et al. Preclinical trials of antitumor therapeutic adenoviral medicine named «Cancerolizin». Voprosy virusologii. 2006;51(6):39-42. (In Russ.)
Nemtsova E, Bezborodova O, Karmakova T, Yakubovskaya R. AdeLact — novel gene-therapy drug: study of detoxifying activity and of recombinant human lactoferrin biodistribution in animal body. Biofarmatsevticheskii zhurnal. 2013;5(6):42-51. (In Russ.)
Alekseenko IV, Chernov IP, Potapov VK, Monastyrskaya GS, Bezborodova OA, Plyutinskaya AD et al. Therapeutic properties of a vector carrying the HSV thymidine kinase and GM-CSF genes and delivered as a complex with a cationic copolymer. J Transl Med. 2015;13(1):78-89. doi:10.1186/s12967-015-0433-0.

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