Current strategies and perspectives in insulin gene therapy for diabetes

References

• Devendra D, Liu E, Eisenbarth GS. Type 1 diabetes: recent developments. Br. Med. J. 328(7442), 750–754 (2004).
   PubMed Web of Science ®Google Scholar
• Butler AE, Janson J, Bonner-Weir S, Ritzel R, Rizza RA, Butler PC. β-cell deficit and increased β cell apoptosis in humans with Type 2 diabetes. Diabetes 52(1), 102–110 (2003).
   PubMed Web of Science ®Google Scholar
• Burke GW, Giancio G. Islet cell transplantation: should we use more than one pancreas? Transplant Proc. 34(5), 1925–1926 (2002).
   PubMed Web of Science ®Google Scholar
• Easom RA. β-granule transport and exocytosis. Semin. Cell Dev. Biol. 11(4), 253–266 (2000).
   PubMed Web of Science ®Google Scholar
• Herenu CB, Morel GR, Bellini MJ et al. Gene therapy in the neuroendocrine system. Front. Horm. Res. 35, 135–142 (2006).
   PubMed Web of Science ®Google Scholar
• Motoyoshi S, Shirotani T, Araki E et al. Cellular characterization of pituitary adenoma cell line (AtT20 cell) transfected with insulin, glucose transporter Type 2 (GLUT2) and glucokinase genes: insulin secretion in response to physiological concentrations of glucose. Diabetologia 41(12), 1492–1501 (1998).
   PubMed Web of Science ®Google Scholar
• Wu L, Nicholson W, Wu CY et al. Engineering physiologically regulated insulin secretion in non-β cells by expressing glucagon-like peptide 1 receptor. Gene Ther. 10(19), 1712–1720 (2003).
   PubMed Web of Science ®Google Scholar
• Corbett JA. K cells: a novel target for insulin gene therapy for the prevention of diabetes. Trends Endocrinol. Metab. 12(4), 140–142 (2001).
   PubMed Web of Science ®Google Scholar
• Ramshur EB, Rull TR, Wice BM. Novel insulin/GIP co-producing cell lines provide unexpected insights into Gut K-cell function in vivo. J. Cell. Physiol. 192(3), 339–350 (2002).
   PubMed Web of Science ®Google Scholar
• Cheung AT, Dayanandan B, Lewis JT et al. Glucose-dependent insulin release from genetically engineered K cells. Science 290(5498), 1959–1962 (2000).
   PubMed Web of Science ®Google Scholar
• Grapin-Botton A. Ductal cells in the pancreas. Int. J. Biochem. Cell Biol. 37(3), 504–510 (2005).
   PubMed Web of Science ®Google Scholar
• Shifrin AL, Auricchio A, Yu QC, Wilson J, Raper SE. Adenoviral vector-mediated insulin gene transfer in the mouse pancreas corrects streptozotocin-induced hyperglycemia. Gene Ther. 8(19), 1480–1489 (2001).
   PubMed Web of Science ®Google Scholar
• Wang Z, Zhu T, Rehman KK et al. Widespread and stable pancreatic gene transfer by adeno-associated virus vectors via different routes. Diabetes 55(4), 875–884 (2006).
   PubMed Web of Science ®Google Scholar
• Bonner-Weir S, Weir GS. New sources of pancreatic β-cells. Nat. Biotechnol. 23(7), 857–861 (2005).
   PubMed Web of Science ®Google Scholar
• Thule PM, Liu JM. Regulated hepatic insulin gene therapy of STZ-diabetic rats. Gene Ther. 7(20), 1744–1752 (2000).
   PubMed Web of Science ®Google Scholar
• Shaw JA, Delday MI, Hart AW, Docherty HM, Maltin CA, Docherty K. Secretion of bioactive human insulin following plasmid-mediated gene transfer to non-neuroendocrine cell lines, primary cultures and rat skeletal muscle in vivo. J. Endocrinol. 172(3), 653–672 (2002).
   PubMed Web of Science ®Google Scholar
• Auricchio A, Gao GP, Yu QC et al. Constitutive and regulated expression of processed insulin following in vivo hepatic gene transfer. Gene Ther. 9(14), 963–971 (2002).
   PubMed Web of Science ®Google Scholar
• Alam T, Sollinger HW. Glucose-regulated insulin production in hepatocytes. Transplantation 74(12), 1781–1787 (2002).
   PubMed Web of Science ®Google Scholar
• Chen R, Meseck ML, McEvoy RC, Woo SL. Glucose-stimulated and self-limiting insulin production by glucose 6-phosphatase promoter driven insulin expression in hepatoma cells. Gene Ther. 7(21), 1802–1809 (2000).
   PubMed Web of Science ®Google Scholar
• Chen R, Meseck ML, Woo SL. Auto-regulated hepatic insulin gene expression in Type 1 diabetic rats. Mol. Ther. 3(4), 584–590 (2001).
   PubMed Web of Science ®Google Scholar
• Lee HC, Kim SJ, Kim KS, Shin HC, Yoon JW. Remission in models of Type 1 diabetes by gene therapy using a single-chain insulin analogue. Nature 408(6811), 483–488 (2000).
   PubMed Web of Science ®Google Scholar
• Wanke IE, Wong NC. Specific problems facing gene therapy for insulin-dependent diabetes mellitus: glucose-regulated insulin secretion from hepatocytes. Proc. West. Pharmacol. Soc. 40, 131–133 (1997).
   PubMedGoogle Scholar
• Olson DE, Paveglio SA, Huey PU, Porter MH, Thule PM. Glucose-responsive hepatic insulin gene therapy of spontaneously diabetic BB/Wor rats. Hum. Gene Ther. 14(15), 1401–1413 (2003).
   PubMed Web of Science ®Google Scholar
• Lee HC. Recent advance in hepatic insulin gene therapy. Diabetes Res. Clin. Pract. 66(Suppl. 1), S3–S10 (2004).
   Google Scholar
• Rivera VM, Wang X, Wardwell S et al. Regulation of protein secretion through controlled aggregation in the endoplasmic reticulum. Science 287(5454), 826–830 (2000).
   PubMed Web of Science ®Google Scholar
• Soria B, Roche E, Berna G, Leon-Quinto T, Reig JA, Martin F. Insulin-secreting cells derived from embryonic stem cells normalize glycemia in streptozotocin-induced diabetic mice. Diabetes 49(2), 157–162 (2000).
   PubMed Web of Science ®Google Scholar
• Chen X, Patil JG, Lok SH, Kon OL. Human liver-derived cells stably modified for regulated proinsulin secretion function as bioimplants in vivo. J. Gene Med. 4(4), 447–458 (2002).
   PubMed Web of Science ®Google Scholar
• Xu R, Janson CG, Mastakov M et al. Quantitative comparison of expression with adeno-associated virus (AAV-2) brain-specific gene cassettes. Gene Ther. 8(17), 1323–1332 (2001).
   PubMed Web of Science ®Google Scholar
• Liu M, Ramos-Castaneda J, Arvan P. Role of the connecting peptide in insulin biosynthesis. J. Biol. Chem. 278(17), 14798–14805 (2003).
   PubMed Web of Science ®Google Scholar
• Wahren J, Jornvall H. C-peptide makes a comeback. Diabetes Metab. Res. Rev. 19(5), 345–347 (2003).
   PubMed Web of Science ®Google Scholar
• Simonson GD, Groskreutz DJ, Gorman CM, McDonald MJ. Synthesis and processing of genetically modified human proinsulin by rat myoblast primary cultures. Hum. Gene Ther. 7(1), 71–78 (1996).
   PubMed Web of Science ®Google Scholar
• Gros L, Riu E, Momtoliu L, Ontiverus M, Lebrigand L, Bosch F. Insulin production by engineered muscle cells. Hum. Gene Ther. 10(7), 1207–1217 (1999).
   PubMed Web of Science ®Google Scholar
• Falqui L, Martinenghi S, Severini GM et al. Reversal of diabetes in mice by implantation of human fibroblasts genetically engineered to release mature human insulin. Hum. Gene Ther. 10(11), 1753–1762 (1999).
   PubMed Web of Science ®Google Scholar
• Wilson MO, Scouqall KT, Ratanamart J, McIntyre EA, Shaw JA. Tetracycline-regulated secretion of human (pro)insulin following plasmid-mediated transfection of human muscle. J. Mol. Endocrinol. 34(2), 391–403 (2005).
   PubMed Web of Science ®Google Scholar
• Scougall KT, Shaw JA. Tetracycline-regulated secretion of human insulin in transfected primary myoblasts. Biochem. Biophys. Res. Commun. 304(1), 167–175 (2003).
   PubMed Web of Science ®Google Scholar
• Scougall KT, Maltin CA, Shaw JA. Tetracycline-regulated secretion of human insulin in a transfected non-endocrine cell line. J. Mol. Endocrinol. 30(3), 331–346 (2003).
   PubMed Web of Science ®Google Scholar
• Lu D, Tamemoto H, Shibata H, Saito H. Regulatable production of insulin from primary-cultured hepatocytes: insulin production is up-regulated by glucagon and cAMP and down-regulated by insulin. Gene Ther. 5(7), 888–895 (1998).
   PubMed Web of Science ®Google Scholar
• Qin XY, Shen KT, Song LJ, Zhang X, Han ZG. Regulated production of mature insulin in rat hepatoma cells: insulin production is up-regulated by dexamethasone and down-regulated by insulin. Acta Biochim. Biophys. Sin. 38(2), 89–94 (2006).
   Google Scholar
• Cournarie F, Azzout-Marniche D, Foretz M, Guitchard C, Ferre B, Foufelle F. The inhibitory effect of glucose on phosphoenolpyruvate carboxykinase gene expression in cultured hepatocytes is transcriptional and requires glucose metabolism. FEBS Lett. 460(3), 527–532 (1999).
   PubMed Web of Science ®Google Scholar
• Quinn PG, Yeagley D. Insulin regulation of PEPCK gene expression: a model for rapid and reversible modulation. Curr. Drug Targets Immune Endocr. Metabol. Disord. 5(4), 423–437 (2005).
   PubMedGoogle Scholar
• Olefsky JM. Gene therapy for rats and mice. Nature 408(6811), 420–421 (2000).
   PubMed Web of Science ®Google Scholar
• Paillard F. Insulin-secreting cells for diabetes. Hum. Gene Ther. 10(11), 1741–1742 (1999).
   PubMed Web of Science ®Google Scholar
• Habener JF, Kemp DM, Thomas MK. Transcriptional regulation in pancreatic development. Endocrinology 146(3), 1025–1034 (2005).
   PubMed Web of Science ®Google Scholar
• Melloul D. Transcription factors in islet development and physiology: role of PDX-1 in β-cell function. Ann. NY Acad. Sci. 1014, 28–37 (2004).
   PubMed Web of Science ®Google Scholar
• Ber I, Shternhall K, Perl S et al. Functional, persistent, and extended liver to pancreas transdifferentiation. J. Biol. Chem. 278(34), 31950–31957 (2003).
   PubMed Web of Science ®Google Scholar
• Ferber S, Halkin A, Cohen H et al. Pancreatic and duodenal homeobox gene 1 induces expression of insulin genes in liver and ameliorates streptozotocin-induced hyperglycemia. Nat. Med. 6(5), 568–572 (2000).
   PubMed Web of Science ®Google Scholar
• Sapir T, Shtrernhall K, Meivar-Levy Y et al. Cell-replacement therapy for diabetes: generating functional insulin-producing tissue from adult human liver cells. Proc. Natl Acad. Sci. USA 102(22), 7964–7969 (2005).
   PubMed Web of Science ®Google Scholar
• Koizumi M, Doi R, Toyoda E et al. Hepatic regeneration and enforced PDX-1 expression accelerate transdifferentiation in liver. Surgery 136(2), 449–457 (2004).
   PubMed Web of Science ®Google Scholar
• Kaneto H, Nakatani Y, Miyatsuka M et al. PDX-1/VP16 fusion protein, together with NeuroD or Ngn3, markedly induces insulin gene transcription and ameliorates glucose tolerance. Diabetes 54(4), 1009–1022 (2005).
   PubMed Web of Science ®Google Scholar
• Miyatsuka T, Kaneto H, Kajimoto Y et al. Ectopically expressed PDX-1 in liver initiates endocrine and exocrine pancreas differentiation but causes dysmorphogenesis. Biochem. Biophys. Res. Commun. 310(3), 1017–1025 (2003).
   PubMed Web of Science ®Google Scholar
• Kaneto H, Matsuoka TA, Nakatani Y et al. A crucial role of MafA as a novel therapeutic target for diabetes. J. Biol. Chem. 280(15), 15047–15052 (2005).
   PubMed Web of Science ®Google Scholar
• Rosskamp RH. Long-acting insulin analogues. Diabetes Care 22(Suppl. 2), B109–B113 (1999).
   PubMedGoogle Scholar
• Bolli GB. Rational use of insulin analogues in the treatment of Type 1 diabetes mellitus. Pediatr. Endocrinol. Rev. 1(1), 9–21 (2003).
   PubMedGoogle Scholar
• Wang F, Carabino JM, Vergara CM. Insulin glargine: a systematic review of a long-acting insulin analogue. Clin. Ther. 25(6), 1541–1577 (2003).
   PubMed Web of Science ®Google Scholar
• Thirion C, Larochelle N, Volpers C et al. Strategies for muscle-specific targeting of adenoviral gene transfer vectors. Neuromuscul. Disord. 12(Suppl. 1), S30–S39 (2002).
   Google Scholar
• Jiang Z, Schiedner G, Gilchrist SC, Kochanek S, Clemens PR. CTLA4Ig delivered by high-capacity adenoviral vector induces stable expression of dystrophin in mdx mouse muscle. Gene Ther. 11(19) 1453–1461 (2004).
   PubMed Web of Science ®Google Scholar
• Goncalves MA, van Nierop GP, Tijssen MR et al. Transfer of the full-length dystrophin-coding sequence into muscle cells by a dual high-capacity hybrid viral vector with site-specific integration ability. J. Virol. 79(5), 3146–3162 (2005).
   PubMed Web of Science ®Google Scholar
• Gregorevic P, Blankinship MJ, Allen JM et al. Systemic delivery of genes to striated muscles using adeno-associated viral vectors. Nat. Med. 10(8), 828–834 (2004).
   PubMed Web of Science ®Google Scholar
• Van Linthout S, Madeddu P. Ex vivo gene transfer for improvement of transplanted pancreatic islet viability and function. Curr. Pharm. Des. 11(22), 2927–2940 (2005).
   PubMed Web of Science ®Google Scholar
• Atouf F, Choi Y, Fowler MJ et al. Generation of islet-like hormone-producing cells in vitro from adult human pancreas. Cell Transplant. 14(10), 735–748 (2005).
   PubMed Web of Science ®Google Scholar
• Todorov I, Omori K, Pascual M et al. Generation of human islets through expansion and differentiation of non-islet pancreatic cells discarded (pancreatic discard) after islet isolation. Pancreas 32(2) 130–138 (2006).
   PubMed Web of Science ®Google Scholar
• Lipsett M, Aikin R, Castellarin M et al. Islet neogenesis: a potential therapeutic tool in Type 1 diabetes. Int. J. Biochem. Cell Biol. 38(5–6), 715–720 (2006).
   PubMed Web of Science ®Google Scholar
• Narang AS, Mahato RI. Biological and biomaterial approaches for improved islet transplantation. Pharmacol. Rev. 58(2), 194–243 (2006).
   PubMed Web of Science ®Google Scholar
• Bradley SP, Rastellini C, da Costa MA et al. Gene silencing in the endocrine pancreas mediated by short-interfering RNA. Pancreas 31(4), 373–379 (2005).
   PubMed Web of Science ®Google Scholar
• Hagerkvist R, Mokhtari D, Myers JW, Tengholm A, Welsh N. siRNA produced by recombinant dicer mediates efficient gene silencing in islet cells. Ann. NY Acad. Sci. 1040, 114–122 (2005).
   PubMed Web of Science ®Google Scholar