The Role of Gene Therapy in the Treatments of Type 1 Diabetes Mellitus: a review

Harem Othman Smail

Abstract


The main aims of this review were to understand the roles of gene therapy in the treatment and prevention of type 1 diabetes mellitus and I will discuss a brief history, approaches, vector types with the future of diabetes following clinical use. Type 1 diabetes mellitus is a metabolic condition that is identified by insufficient insulin development due to pancreatic damage to beta cells. Control, long life, and diagnosis of these metabolic disorders have become vital sources for many scientists and researchers. After 2000, the latest approaches to molecular medicine were introduced as one of the possible therapeutic options for diabetes type 1 diagnosis. Many genes have been reported as a clinical trial so that damaged genes can be treated and three main approaches shown about 50 years ago are islet transplantation, β cell regeneration, and insulin gene therapy to cure and prevent diabetes type. Treating diabetes through gene therapy can promise children and adolescents, but more clinical applications are needed to recognize it as a permanent route.

Keywords


Type 1 diabetes mellitus; Gene therapy; β cells; Virus vectors and islet

Full Text:

PDF

References


Ackeifi, C., Wang, P., Karakose, E., Fox, J.E.M., González, B.J., Liu, H., Wilson, J., Swartz, E., Berrouet, C., Li, Y. and Kumar, K., 2020. GLP-1 receptor agonists synergize with DYRK1A inhibitors to potentiate functional human β cell regeneration. Science Translational Medicine, 12(530).

Afzal, S., Fronza, R. and Schmidt, M., 2020. VSeq-Toolkit: Comprehensive computational analysis of viral vectors in gene therapy. Molecular Therapy-Methods & Clinical Development.

Aghazadeh, Y. and Nostro, M.C., 2017. Cell therapy for type 1 diabetes: current and future strategies. Current diabetes reports, 17(6), p.37.

Aguayo-Mazzucato, C. and Bonner-Weir, S., 2010. Stem cell therapy for type 1 diabetes mellitus. Nature Reviews Endocrinology, 6(3), pp.139-148.

Ahrén, B., 2011. The future of incretin‐based therapy: novel avenues—novel targets. Diabetes, Obesity and Metabolism, 13, pp.158-166.

Akirav, E., Kushner, J.A. and Herold, K.C., 2008. β-cell mass and type 1 diabetes: going, going, gone?. Diabetes, 57(11), pp.2883-2888.

Al Ghofaili, K., Fung, M., Ao, Z., Meloche, M., Shapiro, R.J., Warnock, G.L., Elahi, D., Meneilly, G.S. and Thompson, D.M., 2007. Effect of exenatide on β cell function after islet transplantation in type 1 diabetes. Transplantation, 83(1), pp.24-28.

Alleva, D.G., Rezaee, M., Yip, L., Ren, G., Rosenberg, J., Concepcion, W., Escher, A., Shabahang, S. and Thakor, A.S., 2020. Reversal of Hyperglycemia and Suppression of Type 1 Diabetes in the NOD Mouse with Apoptotic DNA Immunotherapy™(ADi™), ADi-100. Biomedicines, 8(3), p.53.

Apaya, M.K., Kuo, T.F., Yang, M.T., Yang, G., Hsiao, C.L., Chang, S.B., Lin, Y. and Yang, W.C., 2020. Phytochemicals as modulators of β-cells and immunity for the therapy of type 1 diabetes: Recent discoveries in pharmacological mechanisms and clinical potential. Pharmacological Research, p.104754.

Arjmand, B., Alavi-Moghadam, S., Payab, M., Goodarzi, P., Hosseini, M.S., Tayanloo-Beik, A., Rezaei-Tavirani, M. and Larijani, B., 2020. GMP-Compliant Adenoviral Vectors for Gene Therapy.

Badr, A.M., Sharkawy, H., Farid, A.A. and El-Deeb, S., 2020. Curcumin induces regeneration of β cells and suppression of phosphorylated-NF-κB in streptozotocin-induced diabetic mice. The Journal of Basic and Applied Zoology, 81, pp.1-15.

Bellin, M.D., Barton, F.B., Heitman, A., Harmon, J.V., Kandaswamy, R., Balamurugan, A.N., Sutherland, D.E.R., Alejandro, R. and Hering, B.J., 2012. Potent induction immunotherapy promotes long‐term insulin independence after islet transplantation in type 1 diabetes. American Journal of Transplantation, 12(6), pp.1576-1583.

Black, L.A. and Zorina, T., 2020. Genetic profile considerations for induction of allogeneic chimerism as a therapeutic approach for type 1 diabetes mellitus. Drug Discovery Today.

Bonner-Weir, S. and Weir, G.C., 2005. New sources of pancreatic β-cells. Nature biotechnology, 23(7), pp.857-861.

Borkar, S.S., Wakodkar, S.B., Raghatate, P.S. and Mahapatra, D.K., Gene Therapy: New Therapeutic approach to Diabetes Mellitus. Gene, 1, p.9.

Bosi, E., Braghi, S., Maffi, P., Scirpoli, M., Bertuzzi, F., Pozza, G., Secchi, A. and Bonifacio, E., 2001. Autoantibody response to islet transplantation in type 1 diabetes. Diabetes, 50(11), pp.2464-2471.

Brandon, E.P., Scott, M.J., Zimmerman, M.C. and D’Amour, K.A., 2020. Pluripotent Stem Cell–Derived Islet Replacement Therapy for Diabetes. In Second Generation Cell and Gene-based Therapies (pp. 157-181). Academic Press.

Bruni, A., Gala-Lopez, B., Pepper, A.R., Abualhassan, N.S. and Shapiro, A.J., 2014. Islet cell transplantation for the treatment of type 1 diabetes: recent advances and future challenges. Diabetes, metabolic syndrome and obesity: targets and therapy, 7, p.211.

Chen, R., Meseck, M.L. and Woo, S.L., 2001. Auto-regulated hepatic insulin gene expression in type 1 diabetic rats. Molecular Therapy, 3(4), pp.584-590.

Cieluch, A., Uruska, A. and Zozulinska-Ziolkiewicz, D., 2020. Can We Prevent Mitochondrial Dysfunction and Diabetic Cardiomyopathy in Type 1 Diabetes Mellitus? Pathophysiology and Treatment Options. International Journal of Molecular Sciences, 21(8), p.2852.

Cobo-Vuilleumier, N. and Gauthier, B.R., 2020. Time for a paradigm shift in treating type 1 diabetes mellitus: coupling inflammation to islet regeneration. Metabolism, 104, p.154137.

De Haan, P., Van Diemen, F.R. and Toscano, M.G., 2020. Viral gene delivery vectors: the next generation medicines for immune-related diseases. Human Vaccines & Immunotherapeutics, pp.1-8.

De Jesus, D.F. and Kulkarni, R.N., 2020. More is better: combinatorial therapy to restore β-cell function in diabetes. Nature Metabolism, 2(2), pp.130-131.

de Kort, H., de Koning, E.J., Rabelink, T.J., Bruijn, J.A. and Bajema, I.M., 2011. Islet transplantation in type 1 diabetes. Bmj, 342.

Desai, S. and Deshmukh, A., 2020. Mapping of Type 1 Diabetes Mellitus. Current Diabetes Reviews, 16(5), pp.438-441.

Donner, T. and Sarkar, S., 2019. Insulin–pharmacology, therapeutic regimens, and principles of intensive insulin therapy. In Endotext [Internet]. MDText. com, Inc.

Dunbar, C.E., High, K.A., Joung, J.K., Kohn, D.B., Ozawa, K. and Sadelain, M., 2018. Gene therapy comes of age. Science, 359(6372).

Emerich, D.F., 2002. Islet transplantation for diabetes: current status and future prospects. Expert Opinion on Biological Therapy, 2(8), pp.793-803.

Escors, D. and Breckpot, K., 2010. Lentiviral vectors in gene therapy: their current status and future potential. Archivum immunologiae et therapiae experimentalis, 58(2), pp.107-119.

Fang, Q., Zhai, M., Wu, S., Hu, X., Hua, Z., Sun, H., Guo, J., Zhang, W. and Wang, Z., 2019. Adipocyte-derived stem cell-based gene therapy upon adipogenic differentiation on microcarriers attenuates type 1 diabetes in mice. Stem Cell Research & Therapy, 10(1), pp.1-11.

Farooq, T., Rehman, K., Hameed, A. and Akash, M.S.H., 2019. Stem cell therapy and type 1 diabetes mellitus: treatment strategies and future perspectives. In Tissue Engineering and Regenerative Medicine (pp. 95-107). Springer International Publishing.

Friedmann, T., 1992. A brief history of gene therapy. Nature genetics, 2(2), pp.93-98.

Froud, T., Ricordi, C., Baidal, D.A., Hafiz, M.M., Ponte, G., Cure, P., Pileggi, A., Poggioli, R., Ichii, H., Khan, A. and Ferreira, J.V., 2005. Islet transplantation in type 1 diabetes mellitus using cultured islets and steroid‐free immunosuppression: Miami experience. American Journal of Transplantation, 5(8), pp.2037-2046.

Fujikawa, T., Chuang, J.C., Sakata, I., Ramadori, G. and Coppari, R., 2010. Leptin therapy improves insulin-deficient type 1 diabetes by CNS-dependent mechanisms in mice. Proceedings of the National Academy of Sciences, 107(40), pp.17391-17396.

Ginn, S.L., Amaya, A.K., Alexander, I.E., Edelstein, M. and Abedi, M.R., 2018. Gene therapy clinical trials worldwide to 2017: An update. The journal of gene medicine, 20(5), p.e3015.

Goins, W.F., Huang, S., Hall, B., Marzulli, M., Cohen, J.B. and Glorioso, J.C., 2020. Engineering HSV-1 Vectors for Gene Therapy. In Herpes Simplex Virus (pp. 73-90). Humana, New York, NY.

Guney, M.A., Lorberbaum, D.S. and Sussel, L., 2020. Pancreatic β cell regeneration: to β or not to β. Current Opinion in Physiology, 14, pp.13-20.

Handorf, A.M., Sollinger, H.W. and Alam, T., 2015. Insulin gene therapy for type 1 diabetes mellitus. Exp clin transplant, 13(Suppl 1), pp.37-45.

Jeong, Y., Park, J.K.H. and Eun, S., 2020, May. Viral Vector Mediated Interleukin-10 Gene Transfer in Skin Allograft. In Transplantation Proceedings. Elsevier.

Kanao-Kanda, M., Kanda, H., Liu, S., Roy, S., Toborek, M. and Hao, S., 2020. Viral Vector–Mediated Gene Transfer of Glutamic Acid Decarboxylase for Chronic Pain Treatment: A Literature Review. Human gene therapy, 31(7-8), pp.405-414.

Kato, N., Nemoto, K., Nakanishi, K., Morishita, R., Kaneda, Y., Uenoyama, M., Ikeda, T. and Fujikawa, K., 2005. Nonviral gene transfer of human hepatocyte growth factor improves streptozotocin-induced diabetic neuropathy in rats. Diabetes, 54(3), pp.846-854.

Kaur, K.K., 2020. Attempting Getting Insulin Independent Immunotherapies in Type 1 Diabetes Mellitus (T1D) in the Pre Stage 1 (Before Islet Autoantibodies). Acta Scientific Paediatrics, 3, pp.01-04.

Kharroubi, A.T. and Darwish, H.M., 2015. Diabetes mellitus: The epidemic of the century. World journal of diabetes, 6(6), p.850.

Kolodka, T.M., Finegold, M., Moss, L. and Woo, S.L., 1995. Gene therapy for diabetes mellitus in rats by hepatic expression of insulin. Proceedings of the National Academy of Sciences, 92(8), pp.3293-3297.

Lakhtakia, R., 2013. The history of diabetes mellitus. Sultan Qaboos University Medical Journal, 13(3), p.368.

Latres, E., Finan, D.A., Greenstein, J.L., Kowalski, A. and Kieffer, T.J., 2019. Navigating two roads to glucose normalization in diabetes: automated insulin delivery devices and cell therapy. Cell metabolism, 29(3), pp.545-563.

Le Bagge, S., Fotheringham, A.K., Leung, S.S. and Forbes, J.M., 2020. Targeting the receptor for advanced glycation end products (RAGE) in type 1 diabetes. Medicinal Research Reviews.

Lee, H.C., Kim, S.J., Kim, K.S., Shin, H.C. and Yoon, J.W., 2000. Remission in models of type 1 diabetes by gene therapy using a single-chain insulin analogue. Nature, 408(6811), pp.483-488.

Liu, B., Xiang, Y., Liu, Z. and Zhou, Z., 2020. Past, present and future of latent autoimmune diabetes in adults. Diabetes/metabolism research and reviews, 36(1), p.e3205.

Lu, Y., Tang, M., Wasserfall, C., Kou, Z., Campbell-Thompson, M., Gardemann, T., Crawford, J., Atkinson, M. and Song, S., 2006. α 1-Antitrypsin gene therapy modulates cellular immunity and efficiently prevents type 1 diabetes in nonobese diabetic mice. Human gene therapy, 17(6), pp.625-634.

Maanaoui, M., Chetboun, M., Kerr-Conte, J., Gmyr, V., Hubert, T., Caiazzo, R., Jannin, A., Le Mapihan, K., Vantyghem, M.C., Hazzan, M. and Pattou, F., 2020. P1672 IMPACT OF ISLET TRANSPLANTATION VERSUS INSULIN ALONE AFTER KIDNEY TRANSPLANTATION IN TYPE 1 DIABETES PATIENTS. Nephrology Dialysis Transplantation, 35(Supplement_3), pp.gfaa142-P1672.

Maffi, P., Lundgren, T., Tufveson, G., Rafael, E., Shaw, J.A., Liew, A., Saudek, F., Witkowski, P., Golab, K., Bertuzzi, F. and Gustafsson, B., 2020. Targeting CXCR1/2 does not improve insulin secretion after pancreatic islet transplantation: a phase 3, double-blind, randomized, placebo-controlled trial in type 1 diabetes. Diabetes Care, 43(4), pp.710-718.

Matsumoto, S., 2010. Islet cell transplantation for Type 1 diabetes. Journal of diabetes, 2(1), pp.16-22.

Maxwell, K.G., Augsornworawat, P., Velazco-Cruz, L., Kim, M.H., Asada, R., Hogrebe, N.J., Morikawa, S., Urano, F. and Millman, J.R., 2020. Gene-edited human stem cell–derived β cells from a patient with monogenic diabetes reverse preexisting diabetes in mice. Science Translational Medicine, 12(540).

Memon, B. and Abdelalim, E.M., 2020. Stem cell therapy for diabetes: Beta cells versus pancreatic progenitors. Cells, 9(2), p.283.

Naldini, L., 2015. Gene therapy returns to centre stage. Nature, 526(7573), pp.351-360.

Niu, L., Xu, Y.C., Dai, Z. and Tang, H.Q., 2008. Gene therapy for type 1 diabetes mellitus in rats by gastrointestinal administration of chitosan nanoparticles containing human insulin gene. World journal of gastroenterology: WJG, 14(26), p.4209.

Onuma, H., Kawamura, R., Tabara, Y., Yamashita, M., Ohashi, J., Kawasaki, E., Imagawa, A., Yamada, Y., Chujo, D., Takahashi, K. and Suehiro, T., 2019. Variants in the BACH2 and CLEC16A gene might be associated with susceptibility to insulin‐triggered type 1 diabetes. Journal of Diabetes Investigation, 10(6), pp.1447-1453.

Paty, B.W., Ryan, E.A., Shapiro, A.J., Lakey, J.R. and Robertson, R.P., 2002. Intrahepatic islet transplantation in type 1 diabetic patients does not restore hypoglycemic hormonal counterregulation or symptom recognition after insulin independence. Diabetes, 51(12), pp.3428-3434.

Prud'Homme, G.J., Draghia-Akli, R. and Wang, Q., 2007. Plasmid-based gene therapy of diabetes mellitus. Gene therapy, 14(7), pp.553-564.

Ramezani, K., Gheflat, S., Jajarmi, V., Bandehpour, M. and Kazemi, B., 2019. The Study on Possible Gene Therapy on Diabetics Type I Using Insulin Gene under Control of Heat Shock Promoter in Laboratory Animals. Novelty in Biomedicine, 7(2), pp.45-48.

Rickels, M.R., Schutta, M.H., Markmann, J.F., Barker, C.F., Naji, A. and Teff, K.L., 2005. β-Cell function following human islet transplantation for type 1 diabetes. Diabetes, 54(1), pp.100-106.

Rosado-Olivieri, E.A., Aigha, I.I., Kenty, J.H. and Melton, D.A., 2020. Identification of a LIF-Responsive, Replication-Competent Subpopulation of Human β Cells. Cell Metabolism, 31(2), pp.327-338.

Sachs, S., Bastidas-Ponce, A., Tritschler, S., Bakhti, M., Böttcher, A., Sánchez-Garrido, M.A., Tarquis-Medina, M., Kleinert, M., Fischer, K., Jall, S. and Harger, A., 2020. Targeted pharmacological therapy restores β-cell function for diabetes remission. Nature Metabolism, 2(2), pp.192-209.

Samson, S.L. and Chan, L., 2006. Gene therapy for diabetes: reinventing the islet. Trends in Endocrinology & Metabolism, 17(3), pp.92-100.

Scharp, D.W. and Marchetti, P., 2014. Encapsulated islets for diabetes therapy: history, current progress, and critical issues requiring solution. Advanced drug delivery reviews, 67, pp.35-73.

Shapiro, A.J., 2012. Islet transplantation in type 1 diabetes: ongoing challenges, refined procedures, and long-term outcome. The review of diabetic studies: RDS, 9(4), p.385.

Shirley, J.L., de Jong, Y.P., Terhorst, C. and Herzog, R.W., 2020. Immune responses to viral gene therapy vectors. Molecular Therapy, 28(3), pp.709-722.

Simeonovic, C.J., Popp, S.K., Brown, D.J., Li, F.J., Lafferty, A.R., Freeman, C. and Parish, C.R., 2020. Heparanase and Type 1 Diabetes. In Heparanase (pp. 607-630). Springer, Cham.

Sluch, V., Swain, D., Whipple, W., Liao, M., Bhoumik, A., Agulnick, A.D. and Rezania, A., 2019, September. CRISPR-editing of hESCs allows for production of immune evasive cells capable of differentiation to pancreatic progenitors for future type 1 diabetes therapy. In DIABETOLOGIA (Vol. 62, pp. S6-S7). 233 SPRING ST, NEW YORK, NY 10013 USA: SPRINGER.

Smail, H., Kareem, S. and Abdulkareem, N. (2019) “Comparative Study of the Fingerprint Pattern among Diabetic (Type 1) & Non-Diabetic Children in Koya City”, Journal of Advanced Laboratory Research in Biology, 10(2), pp. 41-47.

Takei, I. and Kasatani, T., 2004. Future therapy of diabetes mellitus. Biomedicine & pharmacotherapy, 58(10), pp.578-581.

Teta, M., Rankin, M.M., Long, S.Y., Stein, G.M. and Kushner, J.A., 2007. Growth and regeneration of adult β cells does not involve specialized progenitors. Developmental cell, 12(5), pp.817-826.

Thorel, F., Népote, V., Avril, I., Kohno, K., Desgraz, R., Chera, S. and Herrera, P.L., 2010. Conversion of adult pancreatic α-cells to β-cells after extreme β-cell loss. Nature, 464(7292), pp.1149-1154.

Thyssen, S., Arany, E. and Hill, D.J., 2006. Ontogeny of regeneration of β-cells in the neonatal rat after treatment with streptozotocin. Endocrinology, 147(5), pp.2346-2356.

Tian, C., Ansari, M.J.I., Paez-Cortez, J., Bagley, J., Godwin, J., Donnarumma, M., Sayegh, M.H. and Iacomini, J., 2007. Induction of robust diabetes resistance and prevention of recurrent type 1 diabetes following islet transplantation by gene therapy. The Journal of Immunology, 179(10), pp.6762-6769.

Van Belle, T.L., Coppieters, K.T. and Von Herrath, M.G., 2011. Type 1 diabetes: etiology, immunology, and therapeutic strategies. Physiological reviews, 91(1), pp.79-118.

Verma, I.M., Naldini, L., Kafri, T., Miyoshi, H., Takahashi, M., Blömer, U., Somia, N., Wang, L. and Gage, F.H., 2000. Gene therapy: promises, problems and prospects. In Genes and Resistance to Disease (pp. 147-157). Springer, Berlin, Heidelberg.

Wainwright, S.P., Williams, C., Michael, M., Farsides, B. and Cribb, A., 2006. From bench to bedside? Biomedical scientists’ expectations of stem cell science as a future therapy for diabetes. Social science & medicine, 63(8), pp.2052-2064.

Wang, H., Wei, X., Shi, W., He, J. and Luo, L., 2020. Key Developmental Regulators Suggest Multiple Origins of Pancreatic Beta Cell Regeneration. Zebrafish.

Wang, J.H., Roberts, G.E. and Liu, G.S., 2020. Updates on Gene Therapy for Diabetic Retinopathy. Current Diabetes Reports, 20, pp.1-12.

Wang, M.Y., Chen, L., Clark, G.O., Lee, Y., Stevens, R.D., Ilkayeva, O.R., Wenner, B.R., Bain, J.R., Charron, M.J., Newgard, C.B. and Unger, R.H., 2010. Leptin therapy in insulin-deficient type I diabetes. Proceedings of the national academy of Sciences, 107(11), pp.4813-4819.

Wirth, T., Parker, N. and Ylä-Herttuala, S., 2013. History of gene therapy. Gene, 525(2), pp.162-169.

Wolff, J.A. and Lederberg, J., 1994. An early history of gene transfer and therapy. Human Gene Therapy, 5(4), pp.469-480.

Wong, M.S., Hawthorne, W.J. and Manolios, N., 2010. Gene therapy in diabetes. Self/nonself, 1(3), pp.165-175.

Yi, Z., Waseem Ghani, M., Ghani, H., Jiang, W., Waseem Birmani, M., Ye, L., Bin, L., Cun, L.G., Lilong, A. and Mei, X., 2020. Gimmicks of gamma‐aminobutyric acid (GABA) in pancreatic β‐cell regeneration through transdifferentiation of pancreatic α‐to β‐cells. Cell Biology International, 44(4), pp.926-936.

Yu, X.X. and Xu, C.R., 2020. Understanding generation and regeneration of pancreatic β cells from a single-cell perspective. Development, 147(7).

Zarinsefat, A. and Stock, P.G., 2020. Islet vs pancreas transplantation in nonuremic patients with type 1 diabetes. In Transplantation, Bioengineering, and Regeneration of the Endocrine Pancreas (pp. 417-423). Academic Press.

Zhang, J. and Liu, F., 2020, January. The De-, Re-, and trans-differentiation of β-cells: Regulation and function. In Seminars in Cell & Developmental Biology. Academic Press.

Zhou, Q., Brown, J., Kanarek, A., Rajagopal, J. and Melton, D.A., 2008. In vivo reprogramming of adult pancreatic exocrine cells to β-cells. nature, 455(7213), pp.627-632.

Zhou, X., Zhang, S., Yu, F., Zhao, G., Geng, S., Yu, W., Wang, X.Y. and Wang, B., 2020. Tolerogenic vaccine composited with islet-derived multipeptides and cyclosporin A induces pTreg and prevents Type 1 diabetes in murine model. Human vaccines & immunotherapeutics, 16(2), pp.240-250.




DOI: https://doi.org/10.14421/biomedich.2020.92.57-64

Refbacks



Bookmark and Share


Copyright (c) 2020 Harem Othman Smail



Biology, Medicine, & Natural Product Chemistry
ISSN 2089-6514 (paper) - ISSN 2540-9328 (online)
Published by Sunan Kalijaga State Islamic University & Society for Indonesian Biodiversity.

CC BY NC
This work is licensed under a CC BY-NC