Localized gene expression following administration of adeno-associated viral vectors via pancreatic ducts

Molecular Therapy

Volumn 12, Issue 3, 2005, Pages 519-527

  Loiler, Scott A ^a,b   Tang, Qiushi ^a,b   Clarke, Tracy ^a   Campbell Thompson, Martha L ^a   Chiodo, Vince ^a,b   Hauswirth, William ^a,b   Cruz, Pedro ^a,b   Perret Gentil, Marcel ^b   Atkinson, Mark A ^a   Ramiya, Vijayakumar K ^b   Flotte, Terence R ^a,b  

^a UNIVERSITY OF FLORIDA   (United States)

^b UNIVERSITY OF FLORIDA COLLEGE OF MEDICINE   (United States)

Author keywords

1 antitrypsin; Adeno associated virus; Gene therapy; Pancreas; Targeting

Indexed keywords

ALPHA 1 ANTITRYPSIN; BETA ACTIN; INSULIN; PARVOVIRUS VECTOR; PLASMID VECTOR;

ACINAR CELL; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; AUTOIMMUNITY; CELL REGENERATION; DRUG EFFICACY; DRUG SAFETY; DRUG TARGETING; ENDOSCOPIC RETROGRADE CHOLANGIOPANCREATOGRAPHY; FEMALE; GENE EXPRESSION; GENE LOCATION; HUMAN; HUMAN CELL; IMMUNOHISTOCHEMISTRY; IN VIVO STUDY; INSULIN DEPENDENT DIABETES MELLITUS; MOUSE; MOUSE STRAIN; NONHUMAN; NONVIRAL GENE THERAPY; PANCREAS DUCT; PANCREAS ISLET BETA CELL; RAT; RAT STRAIN; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; VIRUS EXPRESSION;

ALPHA 1-ANTITRYPSIN; ANIMALS; DEPENDOVIRUS; ENZYME-LINKED IMMUNOSORBENT ASSAY; FEMALE; GENE EXPRESSION REGULATION; GENE THERAPY; GENETIC VECTORS; IMMUNOHISTOCHEMISTRY; INSULIN; INSULIN-SECRETING CELLS; MICE; MICE, INBRED C57BL; PANCREAS; PANCREATIC DUCTS; PLASMIDS; PROMOTER REGIONS (GENETICS); RATS; RATS, INBRED F344; REVERSE TRANSCRIPTASE POLYMERASE CHAIN REACTION; RNA, MESSENGER; TIME FACTORS; TISSUE DISTRIBUTION; TRANSGENES;

ADENO-ASSOCIATED VIRUS; RODENTIA;

EID: 23844508678     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ymthe.2005.04.017     Document Type: Article

References (32)

1. B.R. Burkhardt et al. Glucose-responsive expression of the human insulin promoter in HepG2 human hepatoma cells Ann. N.Y. Acad. Sci. 1005 2003 237-241
2. H. Edlund Pancreatic organogenesis - developmental mechanisms and implications for therapy Nat. Rev. Genet. 3 2002 524-532
3. F. Homo-Delarche and H.A. Drexhage Immune cells, pancreas development, regeneration and type 1 diabetes Trends Immunol. 25 2004 222-229
4. K.A. Johansson and A. Grapin-Botton Development and diseases of the pancreas Clin. Genet. 62 2002 14-23
5. A.B. Lowenfels and P. Maisonneuve Epidemiology and prevention of pancreatic cancer Jpn. J. Clin. Oncol. 34 2004 238-244
6. D.S. Michaud Epidemiology of pancreatic cancer Minerva Chir. 59 2004 99-111
7. M.S. Roy R. Klein B.J. O'Colmain B.E. Klein S.E. Moss and J.H. Kempen The prevalence of diabetic retinopathy among adult type 1 diabetic persons in the United States Arch. Ophthalmol. 122 2004 546-551
8. Virgilis, D., [Cystic fibrosis from the exocrine pancreatic point of view]. Harefuah, 143: 67-72, 83.
9. E. Ayuso et al. In vivo gene transfer to pancreatic beta cells by systemic delivery of adenoviral vectors Hum. Gene Ther. 15 2004 805-812
10. A.Y. Wang P.D. Peng A. Ehrhardt T.A. Storm and M.A. Kay Comparison of adenoviral and adeno-associated viral vectors for pancreatic gene delivery in vivo Hum. Gene Ther. 15 2004 405-413
11. H. Taniguchi E. Yamato F. Tashiro H. Ikegami T. Ogihara and J. Miyazaki Beta-cell neogenesis induced by adenovirus-mediated gene delivery of transcription factor pdx-1 into mouse pancreas Gene Ther. 10 2003 15-23
12. T. Flotte et al. Efficient ex vivo transduction of pancreatic islet cells with recombinant adeno-associated virus vectors Diabetes 50 2001 515-520
13. S.A. Loiler et al. Targeting recombinant adeno-associated virus vectors to enhance gene transfer to pancreatic islets and liver Gene Ther. 10 2003 1551-1558
14. R.M. Schmid et al. Liposome mediated in vivo gene transfer into different tissues of the gastrointestinal tract Z. Gastroenterol. 32 1994 665-670
15. Y. Yang S.E. Raper J.A. Cohn J.F. Engelhardt and J.M. Wilson An approach for treating the hepatobiliary disease of cystic fibrosis by somatic gene transfer Proc. Natl. Acad. Sci. USA 90 1993 4601-4605
16. S.E. Raper and R.P. DeMatteo Adenovirus-mediated in vivo gene transfer and expression in normal rat pancreas Pancreas 12 1996 401-410
17. M.D. Walker C.W. Park A. Rosen and A. Aronheim A cDNA from a mouse pancreatic beta cell encoding a putative transcription factor of the insulin gene Nucleic Acids Res. 18 1990 1159-1166
18. R. Mueller T. Krahl and N. Sarvetnick Pancreatic expression of interleukin-4 abrogates insulitis and autoimmune diabetes in nonobese diabetic (NOD) mice J. Exp. Med. 184 1996 1093-1099
19. S. Song et al. Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors Proc. Natl. Acad. Sci. USA 95 1998 14384-14388
20. L. Xu et al CMV-beta-actin promoter directs higher expression from an adeno-associated viral vector in the liver than the cytomegalovirus or elongation factor 1 alpha promoter and results in therapeutic levels of human factor X in mice Hum. Gene Ther. 12 2001 563-573
21. L. Cordier et al. Muscle-specific promoters may be necessary for adeno-associated virus-mediated gene transfer in the treatment of muscular dystrophies Hum. Gene Ther. 12 2001 205-215
22. E.E. Zolotarskaia E.M. Dukhovnaia and R.S. Dreizin Study of the possibilities of inactivating adeno-associated virus type 4 Vopr. Virusol. 3 1975 341-344
23. F.B. Johnson and A.S. Bodily Effect of environmental pH on adenovirus-associated virus Proc. Soc. Exp. Biol. Med. 150 1975 585-590
24. T. Noro et al. Adeno-associated viral vector-mediated expression of endostatin inhibits tumor growth and metastasis in an orthotropic pancreatic cancer model in hamsters Cancer Res. 64 2004 7486-7490
25. K. Goudy et al. Adeno-associated virus vector-mediated IL-10 gene delivery prevents type 1 diabetes in NOD mice Proc. Natl. Acad. Sci. USA 98 2001 13913-13918
26. M. George E. Ayuso A. Casellas C. Costa J.C. Devedjian and F. Bosch Beta cell expression of IGF-I leads to recovery from type 1 diabetes J. Clin. Invest. 109 2002 1153-1163
27. P.L. Brubaker and D.J. Drucker Glucagon-like peptides regulate cell proliferation and apoptosis in the pancreas, gut, and central nervous system Endocrinology 145 2004 2653-2659
28. S.A. Loiler and T. Flotte Alternative capsid strategies for targeting recombinant AAV gene therapy Gene Ther. Regul. 2 2003 139-148
29. D. Grimm A. Kern K. Rittner and J.A. Kleinschmidt Novel tools for production and purification of recombinant adenoassociated virus vectors Hum. Gene Ther. 9 1998 2745-2760
30. S. Zolotukhin et al. Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield Gene Ther. 6 1999 973-985
31. S. Zolotukhin et al. Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors Methods 28 2002 158-167
32. G.P. Gao M.R. Alvira L. Wang R. Calcedo J. Johnston and J.M. Wilson Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy Proc. Natl. Acad. Sci. USA 99 2002 11854-11859