Efficient and persistent transduction of exocrine and endocrine pancreas by adeno-associated virus type 8

Journal of Biomedical Science

Volumn 14, Issue 5, 2007, Pages 585-594

  Cheng, Henrique ^a   Wolfe, Stephanie H ^b   Valencia, Valery ^b   Qian, Keping ^c,d   Shen, Leping ^c,d   Phillips, M Ian ^c,d   Chang, Lung Ji ^d   Zhang, Y Clare ^b  

^a LOUISIANA STATE UNIVERSITY   (United States)

^b UNIVERSITY OF SOUTH FLORIDA   (United States)

^c KECK GRADUATE INSTITUTE   (United States)

^d UNIVERSITY OF FLORIDA   (United States)

Author keywords

cells; Adeno associated virus; Gene therapy; Lentivirus; Pancreas

Indexed keywords

GLUCOSE; GREEN FLUORESCENT PROTEIN; VIRUS VECTOR;

ACINAR CELL; ADENO ASSOCIATED VIRUS; ADENO ASSOCIATED VIRUS TYPE 1; ADENO ASSOCIATED VIRUS TYPE 2; ADENO ASSOCIATED VIRUS TYPE 5; ADENO ASSOCIATED VIRUS TYPE 8; ANALYTIC METHOD; ANIMAL EXPERIMENT; ARTICLE; CELL INFILTRATION; COMPARATIVE STUDY; CONTROLLED STUDY; DOSE RESPONSE; GENE TRANSFER; GLUCOSE BLOOD LEVEL; LENTIVIRINAE; LEUKOCYTE; MALE; MOUSE; NONHUMAN; PANCREAS; PANCREAS DUCT; PANCREAS ISLET; PRIORITY JOURNAL; VIRAL GENE DELIVERY SYSTEM;

ANIMALS; BLOOD GLUCOSE; DEPENDOVIRUS; GENE THERAPY; GENETIC VECTORS; GREEN FLUORESCENT PROTEINS; ISLETS OF LANGERHANS; MICE; MICE, TRANSGENIC; PANCREAS, EXOCRINE; TRANSDUCTION, GENETIC;

ADENO-ASSOCIATED VIRUS; LENTIVIRUS;

EID: 34548664862     PISSN: 10217770     EISSN: 14230127     Source Type: Journal    
DOI: 10.1007/s11373-007-9159-1     Document Type: Article

References (40)

1. Raper S.E., DeMatteo R.P.: Adenovirus-mediated in vivo gene transfer and expression in normal rat pancreas. Pancreas 12:401-410, 1996
2. Ayuso E., Chillon M., Agudo J., Haurigot V., Bosch A., Carretero A., Otaegui P.J., Bosch F.: In vivo gene transfer to pancreatic beta cells by systemic delivery of adenoviral vectors. Hum. Gene Ther. 15:805-812, 2004
3. Shifrin A.L., Auricchio A., Yu Q.C., Wilson, J., Raper S.E.: Adenoviral vector-mediated insulin gene transfer in the mouse pancreas corrects streptozotocin-induced hyperglycemia. Gene Ther. 8:1480-1489, 2001
4. Sigalla J., David A., Anegon I., Fiche M., Huvelin J.M., Boeffard F., Cassard A., Soulillou J.P., Le Mauff B.: Adenovirus-mediated gene transfer into isolated mouse adult pancreatic islets: normal beta-cell function despite induction of an anti-adenovirus immune response. Hum. Gene Ther. 8:1625-1634, 1997
5. McClane S.J., Chirmule N., Burke C.V., Raper S.E.: Characterization of the immune response after local delivery of recombinant adenovirus in murine pancreas and successful strategies for readministration. Hum. Gene Ther. 8:2207-2216, 1997
6. Castaing M., Guerci A., Mallet J., Czernichow P., Ravassard P., Scharfmann R.: Efficient restricted gene expression in beta cells by lentivirus-mediated gene transfer into pancreatic stem/progenitor cells. Diabetologia 48:709-719, 2005
7. Kobinger G.P., Deng S., Louboutin J.P., Vatamaniuk M., Matschinsky F., Markmann J.F., Raper S.E., Wilson J.M.: Transduction of human islets with pseudotyped lentiviral vectors. Hum. Gene Ther. 15:211-219, 2004
8. Giannoukakis N., Mi Z., Gambotto A., Eramo A., Ricordi C., Trucco M., Robbins P.: Infection of intact human islets by a lentiviral vector. Gene Ther. 6:1545-1551, 1999
9. Gallichan W.S., Kafri T., Krahl T., Verma I.M., Sarvetnick N.: Lentivirus-mediated transduction of islet grafts with interleukin 4 results in sustained gene expression and protection from insulitis. Hum. Gene Ther. 9:2717-2726: 1998
10. Carter P.J., Samulski R.J.: Adeno-associated viral vectors as gene delivery vehicles. Int. J. Mol. Med. 6:17-27, 2000
11. Grimm D., Kay M.A.; From virus evolution to vector revolution: use of naturally occurring serotypes of adeno-associated virus (AAV) as novel vectors for human gene therapy. Curr. Gene Ther. 3: 281-304, 2003
12. Flannery J.G., Zolotukhin S., Vaquero M.I., LaVail M.M., Muzyczka N., Hauswirth W.W.; Efficient photoreceptor-targeted gene expression in vivo by recombinant adeno-associated virus. Proc. Natl. Acad. Sci. USA 94: 6916-6921, 1997
13. Song S., Morgan M., Ellis T., Poirier A., Chesnut K., Wang J., Brantly M., Muzyczka N., Byrne B.J., Atkinson M., Flotte T.R.; Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors. Proc. Natl. Acad. Sci. USA 95: 14384-14388, 1998
14. Zhang Y.C., Pileggi A., Agarwal A., Molano R.D., Powers M., Brusko T., Wasserfall C., Goudy K., Zahr E., Poggioli R., Scott-Jorgensen M., Campbell-Thompson M., Crawford J.M., Nick H., Flotte T., Ellis T.M., Ricordi C., Inverardi L., Atkinson M.A.; Adeno-associated virus-mediated IL-10 gene therapy inhibits diabetes recurrence in syngeneic islet cell transplantation of NOD mice. Diabetes 52: 708-716, 2003
15. Flotte T., Agarwal A., Wang J., Song S., Fenjves E.S., Inverardi L., Chesnut K., Afione S., Loiler S., Wasserfall C., Kapturczak M., Ellis T., Nick H., Atkinson M.: Efficient ex vivo transduction of pancreatic islet cells with recombinant adeno-associated virus vectors. Diabetes 50: 515-520, 2001
16. Kapturczak M., Zolotukhin S., Cross J., Pileggi A., Molano R.D., Jorgensen M., Byrne B., Flotte T.R., Ellis T., Inverardi L., Ricordi C., Nick H., Atkinson M., Agarwal A., Transduction of human and mouse pancreatic islet cells using a bicistronic recombinant adeno-associated viral vector. Mol. Ther. 5:154-160, 2002
17. Loiler S.A., Conlon T.J., Song S., Tang Q., Warrington K.H., Agarwal A., Kapturczak M., Li C., Ricordi C., Atkinson M.A., Muzyczka N., Flotte T.R.: Targeting recombinant adeno-associated virus vectors to enhance gene transfer to pancreatic islets and liver. Gene Ther. 10: 1551-1558, 2003
18. Rehman K.K., Wang Z., Bottino R., Balamurugan A.N., Trucco M., Li J., Xiao X., Robbins P.D.: Efficient gene delivery to human and rodent islets with double-stranded (ds) AAV-based vectors. Gene Ther. 12:1313-1323, 2005
19. Wang A.Y., Peng P.D., Ehrhardt A., Storm T.A., Kay M.A.: Comparison of adenoviral and adeno-associated viral vectors for pancreatic gene delivery in vivo. Hum. Gene Ther. 15:405-413, 2004
20. Loiler S.A., Tang Q., Clarke T., Campbell-Thompson M.L., Chiodo V., Hauswirth W., Cruz P., Perret-Gentil M., Atkinson M.A., Ramiya V.K., Flotte T.R.: Localized gene expression following administration of adeno-associated viral vectors via pancreatic ducts. Mol. Ther. 12:519-527, 2005
21. Wang Z., Zhu T., Rehman K.K., Bertera S., Zhang J., Chen C., Papworth G., Watkins S., Trucco M., Robbins P.D., Li J., Xiao X.: Widespread and stable pancreatic gene transfer by adeno-associated virus vectors via different routes. Diabetes 55:875-884, 2006
22. Chang L.J., Zaiss A.K.: Lentiviral vectors. Prepartion and use. Method. Mol. Med. 69:303-318, 2002
23. Chen X.G., Zhu H.Z., Gong J.L., Li F., Xue J.L.: Efficient delivery of human clotting factor IX after injection of lentiviral vectors in utero. Acta Pharmacol. Sin. 25:789-793, 2004
24. Follenzi A., Sabatino G., Lombardo A., Boccaccio C., Naldini L.: Efficient gene delivery and targeted expression to hepatocytes in vivo by improved lentiviral vectors. Hum. Gene Ther. 13:243-260, 2002
25. Nakai H., Fuess S., Storm T.A., Muramatsu S., Nara Y., Kay M.A.: Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice. J. Virol. 79:214-224, 2005
26. Wang Z., Zhu T., Qiao C., Zhou L., Wang B., Zhang J., Chen C., Li J., Xiao X.: Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart. Nat. Biotechnol. 23:321-328, 2005
27. Xiao X., Li J., Samulski R.J.: Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. J. Virol. 70:8098-8108, 1996
28. Sutherland R.M., McKenzie B.S., Zhan Y., Corbett A.J., Fox-Marsh A., Georgiou H.M., Harrison L.C., Lew A.M.: Anti-CD45RB antibody deters xenograft rejection by modulating T cell priming and homing. Int. Immunol. 14:953-962, 2002
29. Akache B., Grimm D., Pandey K., Yant S.R., Xu H., Kay M.A.: The 37/67-kilodalton laminin receptor is a receptor for adeno-associated virus serotypes 8, 2, 3, and 9. J. Virol. 80:9831-9836, 2006
30. Thomas C.E., Storm T.A., Huang Z., Kay M.A.: Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors. J. Virol. 78:3110-3122, 2004
31. Kaplitt M.G., Leone P., Samulski R.J., Xiao X., Pfaff D.W. O'Malley KL, During M.J.: Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain. Nat. Genet. 8:148-154, 1994
32. Snyder R.O., Miao C., Meuse L., Tubb J., Donahue B.A., Lin H.F., Stafford D.W., Patel S., Thompson A.R., Nichols T., Read M.S., Bellinger D.A., Brinkhous K.M., Kay M.A.: Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors. Nat. Med. 5:64-70, 1999
33. Mingozzi F., Schuttrumpf J., Arruda V.R., Liu Y., Liu Y.L. High K.A., Xiao W., Herzog R.W.: Improved hepatic gene transfer by using an adeno-associated virus serotype 5 vector. J. Virol. 76:10497-10502, 2002
34. Davidson B.L., Stein C.S., Heth J.A., Martins I., Kotin R.M., Derksen T.A., Zabner J., Ghodsi A., Chiorini J.A.: Recombinant adeno-associated virus type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian central nervous system. Proc. Natl. Acad. Sci. USA 97:3428-3432, 2000
35. Zabner J., Seiler M., Walters R., Kotin R.M., Fulgeras W., Davidson B.L., Chiorini J.A.: Adeno-associated virus type 5 (AAV5) but not AAV2 binds to the apical surfaces of airway epithelia and facilitates gene transfer. J. Virol. 74:3852-3858, 2000
36. Higashikawa F., Chang L.: Kinetic analyses of stability of simple and complex retroviral vectors. Virology 280:124-131, 2001
37. Chirmule N., Propert K., Magosin S., Qian Y., Qian R., Wilson J.: Immune responses to adenovirus and adeno-associated virus in humans. Gene Ther. 6:1574-1583, 1999
38. Zhang Y.C., Powers M., Wasserfall C., Brusko T., Song S., Flotte T., Snyder R.O., Potter M., Scott-Jorgensen M., Campbell-Thompson M., Crawford J.M., Nick H.S., Agarwal A., Ellis T.M., Atkinson M.A.: Immunity to adeno-associated virus serotype 2 delivered transgenes imparted by genetic predisposition to autoimmunity. Gene Ther. 11:233-240, 2004
39. Gao G.P., Alvira M.R., Wang L., Calcedo R., Johnston J., Wilson J.M.: Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc. Natl. Acad. Sci. USA 99:11854-11859, 2002
40. Wang L., Calcedo R., Nichols T.C., Bellinger D.A., Dillow A., Verma I.M., Wilson J.M.: Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy. Blood 105:3079-3086, 2005