Gene therapy for type 1 diabetes: New approaches

Minerva Medica

Volumn 95, Issue 2, 2004, Pages 93-104

  Morral, N ^a  

^a INDIANA UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

Cell differentiation; Cell division; Diabetes mellitus, insulin dependent; Gene therapy; Promoter regions

Indexed keywords

C PEPTIDE; FURIN; GLUCOSE; GLUCOSE 6 PHOSPHATASE; GLUCOSE TRANSPORTER 2; INSULIN; PARVOVIRUS VECTOR; PHOSPHOENOLPYRUVATE CARBOXYKINASE (GTP); PROINSULIN; PYRUVATE KINASE; RNA; SOMATOMEDIN BINDING PROTEIN 1; SOMATOMEDIN C;

CELL DIFFERENTIATION; DIABETIC NEPHROPATHY; DIABETIC NEUROPATHY; DIABETIC RETINOPATHY; GENE THERAPY; GLUCONEOGENESIS; GLUCOSE BLOOD LEVEL; GLUCOSE OXIDATION; HUMAN; HYPOGLYCEMIA; INSULIN DEPENDENT DIABETES MELLITUS; INSULIN INFUSION; INSULIN SYNTHESIS; NONHUMAN; PANCREAS ISLET BETA CELL; PROMOTER REGION; REVIEW; WEIGHT GAIN;

DIABETES MELLITUS, TYPE 1; GENE THERAPY; GLUCOKINASE; GLUCOSE; HUMANS; INSULIN; INTESTINES; ISLETS OF LANGERHANS; ISLETS OF LANGERHANS TRANSPLANTATION; LIVER; OXIDATION-REDUCTION; PROMOTER REGIONS (GENETICS); PROPROTEIN CONVERTASE 1; PROPROTEIN CONVERTASE 2; REGENERATION;

EID: 3042644926     PISSN: 00264806     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Review

References (60)

1. Murray RK, Granner DK, Mayes PA, Rodwell VW. Harper's biochemistry. Stamford, CT: Appleton & Lange; 2000.
2. The effects of intensive treatment of diabetes on the development and progression of long-term complications on insulin-dependent diabetes mellitus. Diabetes Control and Complications Trial Research Group. N Engl J Med 1993;329:977-88.
3. Hypoglycemia in the Diabetes Control and Complications Trial. The Diabetes Control and Complications Trial Research Group. Diabetes 1997;46:271-86.
4. Muzzin P, Eisensmith RC, Copeland KC, Woo SLC. Hepatic insulin gene expression as treatment for type I diabetes mellitus in rats. Mol Endocrinol 1997;11: 833-7.
5. Hosaka M, Nagahama M, Kim WS, Watanabe T, Hatsuzawa K, Ikemizu J et al. Arg-X-Lys/Arg-Arg motif as a signal for precursor cleavage catalyzed by furin within the constitutive secretory pathway. J Biol Chem 1991;266:12127-30.
6. Yanagita M, Hoshino H, Nakayama K, Takeuchi T. Processing of mutated proinsulin with tetrabasic cleavage sites to mature insulin reflects the expression of furin in nonendocrine cell lines. Endocrinology 1993;133:639-44.
7. Short DK, Okada S, Yamauchi K, Pessin JE. Adenovirus-mediated transfer of a modified human proinsulin gene reverses hyperglycemia in diabetic mice. Am J Physiol 1998;275:E748-56.
8. Thule PM, Liu JM. Regulated hepatic insulin gene therapy of STZ-diabetic rats. Gene Ther 2000;7:1744-52.
9. 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 2000;408:483-8.
10. Dong H, Altomonte J, Morral N, Meseck M, Thung SN, Woo SL. Basal insulin gene expression significantly improves conventional insulin therapy in type 1 diabetic rats. Diabetes 2002;51:130-8.
11. Shenk T. Adenoviridae: the viruses and their replication. In: Fields BN, Knipe DM, Howley PM, Chanock RM, Monath TP, Melnick JL et al. editors. Fields virology. Vol. 2. Philadelphia: Lippincott-Raven Publishers; 1996. p. 2111-48.
12. Herz J, Gerard R. Adenovirus-mediated transfer of low density receptor gene acutely accelerates cholesterol clearance in normal mice. Proc Acad Natl Sci USA 1993;90:2812-6.
13. Stratford-Perricaudet LD, Levrero M, Chase JF, Perricaudet M, Briand P. Evaluation of the transfer and expression in mice of an enzyme-encoding gene using a human adenovirus vector. Hum Gene Ther 1990; 1:241-56.
14. Raskin P, Klaff L, Bergenstal R, Halle JP, Donley D, Mecca T. A 16-week comparison of the novel insulin analog insulin glargine (HOE 901) and NPH human insulin used with insulin lispro in patients with type 1 diabetes. Diabetes Care 2000;23:1666-71.
15. Robertson DG, Marino EM, Thule PM, Seneviratne CK, Murphy LJ. Insulin and glucocorticoids regulate IGFBP1 expression via a common promoter region. Biochem Biophys Res Commun 1994;200:226-32.
16. Gros L, Montoliu L, Riu E, Lebrigand L, Bosch F. Regulated production of mature insulin by non-beta-cells. Hum Gene Ther 1997;8:2249-59.
17. Chen R, Meseck M, McEvoy RC, Woo SL. Glucose-stimulated and self-limiting insulin production by glucose 6-phosphatase promoter driven insulin expression in hepatoma cells. Gene Ther 2000;7:1802-9.
18. Chen R, Meseck ML, Woo SL. Auto-regulated hepatic insulin gene expression in type 1 diabetic rats. Mol Ther 2001;3:584-90.
19. Miao CH, Nakai H, Thompson AR, Storm TA, Chiu W, Snyder RO et al. Nonrandom transduction of recombinant adeno-associated virus vectors in mouse hepatocytes in vivo: cell cycling does not influence hepatocyte transduction. J Virol 2000;74:3793-803.
20. Linden RM, Ward P, Giraud C, Winocour E, Berns KI. Site-specific integration by adeno-associated virus. Proc Natl Acad Sci U S A 1996;93:11288-94.
21. Alam T, Sollinger HW. Glucose-regulated insulin production in hepatocytes. Transplantation 2002;74: 1781-7.
22. Jetton TL, Liang Y, Pettepher CC, Zimmerman EC, Cox FG, Horvath K et al. Analysis of upstream glucokinase promoter activity in transgenic mice and identification of glucokinase in rare neuroendocrine cells in the brain and gut. J Biol Chem 1994;3641-54.
23. Matschinsky FM. A lesson in metabolic regulation inspired by the glucokinase glucose sensor paradigm. Diabetes 1996;45:223-41.
24. Sibrowski W, Seitz HJ. Rapid action of insulin and cyclic AMP in the regulation of functional messenger RNA coding for glucokinase in rat liver. J Biol Chem 1984;259:343-6.
25. Velho G, Froguel P, Clement K, Pueyo ME, Rakotoambinina B, Zouali H et al. Primary pancreatic beta-cell secretory defect caused by mutations in glucokinase gene in kindreds of maturity onset diabetes of the young. Lancet 1992;340:444-8.
26. Froguel P, Zouali H, Vionnet N, Velho G, Vaxiliaire M, Sun F et al. Familial hyperglycemia due to mutations in glucokinase. Definition of a subtype of diabetes mellitus. N Engl J Med 1993;328:697-702.
27. Byrne MM, Sturis J, Clement K, Vionnet N, Pueyo ME, Stoffel M et al. Insulin secretory abnormalities in subjects with hyperglycemia due to glucokinase mutations. J Clin Invest 1994;93:1120-30.
28. Caro JF, Triester S, Patel VK, Tapscott EB, Frazier NL, Dohm GL. Liver glucokinase: decreased activity in patients with type II diabetes. Horm Metab Res 1995;27: 19-22.
29. Hariharan N, Farrelly D, Hagan D, Hillyer D, Arbeeny C, Sabrah T et al. Expression of human hepatic glucokinase in transgenic mice liver results in decreased glucose levels and reduced body weight. Diabetes 1997;46:11-6.
30. Ferre T, Pujol A, Riu E, Bosch F, Valera A. Correction of diabetic alterations by glucokinase. Proc Natl Acad Sci USA 1996;93:7225-30.
31. Morral N, McEvoy R, Dong H, Meseck M, Altomonte J, Thung S et al. Adenovirus-mediated expression of glucokinase in the liver as an adjuvant treatment for type 1 diabetes. Hum Gene Ther 2002;13:1561-70.
32. Otaegui PJ, Ferre T, Pujol A, Riu E, Jimenez R, Bosch F. Expression of glucokinase in skeletal muscle: a new approach to counteract diabetic hyperglycemia. Hum Gene Ther 2000;11:1543-52.
33. Otaegui PJ, Ontiveros M, Ferre T, Riu E, Jimenez R, Bosch F. Glucose-regulated glucose uptake by transplanted muscle cells expressing glucokinase counteracts diabetic hyperglycemia. Hum Gene Ther 2002;13:2125-33.
34. Shapiro AM, Lakey JR, Ryan EA, Korbutt GS, Toth E, Warnock GL et al. Islet transplantation in seven patients with type 1 diabetes mellitus using a glucocorticoid-free immunosuppressive regimen. N Engl J Med 2000;343:230-8.
35. Shapiro AM, Nanji SA, Lakey JR. Clinical islet transplant: current and future directions towards tolerance. Immunol Rev 2003;196:219-36.
36. Ryan EA, Lakey JR, Paty BW, Imes S, Korbutt GS, Kneteman NM et al. Successful islet transplantation: continued insulin reserve provides long-term glycemic control. Diabetes 2002;51:2148-57.
37. Alison M. Liver stem cells: a two compartment system. Curr Opin Cell Biol 1998;10:710-5.
38. Grompe M. Pancreatic-hepatic switches in vivo. Mech Dev 2003;120:99-106.
39. Ferber S, Halkin A, Cohen H, Ber I, Einav Y, Goldberg I et al. Pancreatic and duodenal homeobox gene 1 induces expression of insulin genes in liver and ameliorates streptozotocin-induced hyperglycemia. Nat Med 2000;6:568-72.
40. Ber I, Shternhall K, Perl S, Ohanuna Z, Goldberg I, Barshack I et al. Functional, persistent, and extended liver to pancreas transdifferentiation. J Biol Chem 2003;278:31950-7.
41. Kojima H, Fujimiya M, Matsumura K, Younan P, Imaeda H, Maeda M et al. NeuroD-betacellulin gene therapy induces islet neogenesis in the liver and reverses diabetes in mice. Nat Med 2003;9:596-603.
42. Mitani K, Graham FL, Caskey CT, Kochanek S. Rescue, propagation and partial purification of a helper virus-dependent adenovirus vector. Proc Natl Acad Sci USA 1995;92:3854-8.
43. Clemens PR, Kochanek S, Sunada Y, Chan S, Chen HH, Campbell KP et al. In vivo muscle gene transfer of full-length dystrophin with an adenoviral vector that lacks all viral genes. Gene Ther 1996;3:965-72.
44. Fisher KJ, Choi H, Burda J, Chen S-J, Wilson JM. Recombinant adenovirus deleted of all viral genes for gene therapy of cystic fibrosis. Virology 1996;217:11-22.
45. Haecker SE, Stedman HH, Balice-Gordon RJ, Smith DBJ, Greelish JP, Mitchell MA et al. In vivo expression of full-length human dystrophin from adenoviral vectors deleted of all viral genes. Hum Gene Ther 1996;7:1907-14.
46. Kochanek S, Clemens PR, Mitani K, Chen H-H, Chan S, Caskey CT. A new adenoviral vector: replacement of all viral coding sequences with 28 kb of DNA independently expressing both full-lenght dystrophin and b-galactosidase. Proc Natl Acad Sci USA 1996;93:5731-6.
47. Kumar-Singh R, Chamberlain JS. Encapsidated adenovirus minichromosomes allow delivery and expression of a 14 kb dystrophin cDNA to muscle cells. Hum Mol Genet 1996;5:913-21.
48. Lieber A, He C, Kirillova I, Kay MA. Recombinant adenoviruses with large deletions generated by cre-mediated excision exhibit biological properties compared with first-generation vectors in vitro and in vivo. J Virol 1996;70:8944-60.
49. Parks RJ, Chen L, Anton M, Sankar U, Rudnicki MA, Graham FL. A helper-dependent adenovirus vector system: removal of helper virus by cre-mediated excision of the viral packaging signal. Proc Natl Acad Sci U S A 1996;93:13565-70.
50. Alemany R, Dai Y, Lou YC, Sethi E, Prokopenko E, Josephs SF et al. Complementation of helper-dependent adenoviral vectors: size effects and titer fluctuations. J Virol Methods 1997;68:147-59.
51. Chen HH, Mack LM, Kelly R, Ontell M, Kochanek S, Clemens PR. Persistence in muscle of an adenoviral vector that lacks all viral genes. Proc Natl Acad Sci U S A 1997;94:1645-50.
52. Hardy S, Kitamura M, Harris-Stansil T, Dai Y, Phipps ML. Construction of adenovirus vectors through Cre-lox recombination. J Virol 1997;71:1842-9.
53. Morral N, Parks R, Zhou H, Langston C, Schiedner G, Quinones J et al. High doses of a helper-dependent adenoviral vector yield supraphysiological levels of a1-antitrypsin with negligible toxicity. Hum Gene Ther 1998;9:2709-16.
54. George M, Ayuso E, Casellas A, Costa C, Devedjian JC, Bosch F. Beta cell expression of IGF-I leads to recovery from type 1 diabetes. J Clin Invest 2002;109:1153-63.
55. Le Roith D. Seminars in medicine of the Beth Israel Deaconess Medical Center. Insulin-like growth factors. N Engl J Med 1997;336:633-40.
56. Le Roith D, Butler AA. Insulin-like growth factors in pediatric health and disease. J Clin Endocrinol Metab 1999;84:4355-61.
57. Taniguchi H, Yamato E, Tashiro F, Ikegami H, Ogihara T, Miyazaki J. β-cell neogenesis induced by adenovirus mediated gene delivery of transcription factor pdx-1 into mouse pancreas. Gene Ther 2003;10:15-23.
58. Cheung AT, Dayanandan B, Lewis JT, Korbutt GS, Rajotte RV, Bryer-Ash M et al. Glucose-dependent insulin release from genetically engineered K cells. Science 2000;290:1959-62.
59. Lau C, Soriano HE, Ledley FD, Finegold MJ, Wolfe JH, Birkenmeier EH et al. Retroviral gene transfer into the intestinal epithelium. Hum Gene Ther 1995;6:1145-51.
60. Seppen J, Barry SC, Klinkspoor JH, Katen LJ, Lee SP, Garcia JV et al. Apical gene transfer into quiescent human and canine polarized intestinal epithelial cells by lentivirus vectors. J Virol 2000;74:7642-5.