Improved cardiac gene transfer by transcriptional and transductional targeting of adeno-associated viral vectors

Cardiovascular Research

Volumn 70, Issue 1, 2006, Pages 70-78

  Müller, Oliver J ^a,d   Leuchs, Barbara ^a   Pleger, Sven T ^d   Grimm, Dirk ^b   Franz, Wolfgang M ^c   Katus, Hugo A ^d   Kleinschmidt, Jürgen A ^a  

^a GERMAN CANCER RESEARCH CENTER DKFZ   (Germany)

^b STANFORD UNIVERSITY SCHOOL OF MEDICINE   (United States)

^c LUDWIG MAXIMILIANS UNIVERSITY   (Germany)

^d UNIVERSITY HOSPITAL HEIDELBERG   (Germany)

Author keywords

Gene expression; Gene therapy; Gene transfer; Heart; In vivo; Mouse; Rat

Indexed keywords

MYOSIN LIGHT CHAIN; PROTEOHEPARAN SULFATE;

ADENO ASSOCIATED VIRUS; ARTICLE; CELL SURFACE; CONTROLLED STUDY; CYTOMEGALOVIRUS; ENZYME ACTIVITY; GENE CONSTRUCT; GENE EXPRESSION; GENE TARGETING; GENE TRANSFER; GENETIC TRANSCRIPTION; GENETIC TRANSDUCTION; HEART LEFT VENTRICLE; HUMAN; HUMAN CELL; IN VITRO STUDY; IN VIVO STUDY; INTRAVENOUS DRUG ADMINISTRATION; PARVOVIRUS VECTOR; PRIORITY JOURNAL; REPORTER GENE; SEROTYPE; SYSTEMIC CIRCULATION; VIRUS CAPSID;

ANIMALS; CAPSID; CELLS, CULTURED; CYTOMEGALOVIRUS; DEPENDOVIRUS; FEMALE; GENE TARGETING; GENE THERAPY; GENETIC ENGINEERING; GENETIC VECTORS; HEART DISEASES; HEPARIN; INJECTIONS, INTRAVENOUS; LIVER; LUCIFERASES; MICE; MICE, INBRED STRAINS; MYOCYTES, CARDIAC; MYOSIN LIGHT CHAINS; PROMOTER REGIONS (GENETICS); PROTEOGLYCANS; RATS; RATS, SPRAGUE-DAWLEY; TRANSDUCTION, GENETIC;

EID: 33645064807     PISSN: 00086363     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.cardiores.2005.12.017     Document Type: Article

References (43)

1. E.C. Svensson, D.J. Marshall, K. Woodard, H. Lin, F. Jiang, and L. Chu Efficient and stable transduction of cardiomyocytes after intramyocardial injection or intracoronary perfusion with recombinant adeno-associated virus vectors Circulation 99 1999 201 205
2. L.G. Melo, R. Agrawal, L. Zhang, M. Rezvani, A.A. Mangi, and A. Ehsan Gene therapy strategy for long-term myocardial protection using adeno-associated virus-mediated delivery of heme oxygenase gene Circulation 105 2002 602 607
3. T. Kawada, M. Nakazawa, S. Nakauchi, K. Yamazaki, R. Shimamoto, and M. Urabe Rescue of hereditary form of dilated cardiomyopathy by rAAV-mediated somatic gene therapy: amelioration of morphological findings, sarcolemmal permeability, cardiac performances, and the prognosis of TO-2 hamsters Proc Natl Acad Sci U S A 99 2002 901 906
4. M. Hoshijima, Y. Ikeda, Y. Iwanaga, S. Minamisawa, M.O. Date, and Y. Gu Chronic suppression of heart-failure progression by a pseudophosphorylated mutant of phospholamban via in vivo cardiac rAAV gene delivery Nat Med 8 2002 864 871
5. M.C. LaPointe, X.P. Yang, O.A. Carretero, and Q. He Left ventricular targeting of reporter gene expression in vivo by human BNP promoter in an adenoviral vector Am J Physiol Heart Circ Physiol 283 2002 H1439 H1445
6. T. Rothmann, H.A. Katus, R. Hartong, M. Perricaudet, and W.M. Franz Heart muscle-specific gene expression using replication defective recombinant adenovirus Gene Ther 3 1996 919 926
7. W.M. Franz, T. Rothmann, N. Frey, and H.A. Katus Analysis of tissue-specific gene delivery by recombinant adenoviruses containing cardiac-specific promoters Cardiovasc Res 35 1997 560 566
8. F. Griscelli, P. Gilardi-Hebenstreit, N. Hanania, W.M. Franz, P. Opolon, and M. Perricaudet Heart-specific targeting of beta-galactosidase by the ventricle-specific cardiac myosin light chain 2 promoter using adenovirus vectors Hum Gene Ther 9 1998 1919 1928
9. R. Aikawa, G.S. Huggins, and R.O. Snyder Cardiomyocyte-specific gene expression following recombinant adeno-associated viral vector transduction J Biol Chem 277 2002 18979 18985
10. M.I. Phillips, Y. Tang, K. Schmidt-Ott, K. Qian, and S. Kagiyama Vigilant vector: heart-specific promoter in an adeno-associated virus vector for cardioprotection Hypertension 39 2002 651 655
11. K.J. Lee, R.S. Ross, H.A. Rockman, A.N. Harris, T.X. O'Brien, and M. van Bilsen Myosin light chain-2 luciferase transgenic mice reveal distinct regulatory programs for cardiac and skeletal muscle-specific expression of a single contractile protein gene J Biol Chem 267 1992 15875 15885
12. W.M. Franz, D. Breves, K. Klingel, G. Brem, P.H. Hofschneider, and R. Kandolf Heart-specific targeting of firefly luciferase by the myosin light chain-2 promoter and developmental regulation in transgenic mice Circ Res 73 1993 629 637
13. M. Boshart, F. Weber, G. Jahn, K. Dorsch-Hasler, B. Fleckenstein, and W. Schaffner A very strong enhancer is located upstream of an immediate early gene of human cytomegalovirus Cell 41 1985 521 530
14. N. Frey, W.M. Franz, K. Glöckner, M. Degenhardt, M. Müller, and O. Müller Transgenic rat hearts expressing a human cardiac troponin T deletion reveal diastolic dysfunction and ventricular arrhythmias Cardiovasc Res 47 2000 254 264
15. O.J. Müller, M. Lange, H. Rattunde, H.P. Lorenzen, M. Müller, and N. Frey Transgenic rat hearts overexpressing SERCA2a show improved contractility under baseline conditions and pressure overload Cardiovasc Res 59 2003 380 389
16. A.C. Nathwani, A. Davidoff, H. Hanawa, J.F. Zhou, E.F. Vanin, and A.W. Nienhuis Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA Blood 97 2001 1258 1265
17. C. Summerford, and R.J. Samulski Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions J Virol 72 1998 1438 1445
18. C. Summerford, J.S. Bartlett, and R.J. Samulski AlphaVbeta5 integrin: a co-receptor for adeno-associated virus type 2 infection Nat Med 5 1999 78 82
19. K. Qing, C. Mah, J. Hansen, S. Zhou, V. Dwarki, and A. Srivastava Human fibroblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2 Nat Med 5 1999 71 77
20. A. Kern, K. Schmidt, C. Leder, O.J. Müller, C.E. Wobus, and K. Bettinger Identification of a heparin-binding motif on AAV-2 capsids J Virol 77 2003 11072 11081
21. D. Grimm, M.A. Kay, and J.A. Kleinschmidt Helper virus-free, optically controllable, and two-plasmid-based production of adeno-associated virus vectors of serotypes 1 to 6 Mol Ther 7 2003 839 850
22. D. Grimm, and M.A. Kay 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 2003 281 304
23. 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 U S A 99 2002 11854 11859
24. J.E. Rabinowitz, F. Rolling, C. Li, H. Conrath, W. Xiao, and X. Xiao Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity J Virol 76 2002 791 801
25. S. Zolotukhin, M. Potter, W.W. Hauswirth, J. Guy, and N. Muzyczka A "humanized" green fluorescent protein cDNA adapted for high-level expression in mammalian cells J Virol 70 1996 4646 4654
26. M. Müller, B.K. Fleischmann, S. Selbert, G.J. Ji, E. Endl, and G. Middeler Selection of ventricular-like cardiomyocytes from ES cells in vitro FASEB J 14 2000 2540 2548
27. W.W. Hauswirth, A.S. Lewin, S. Zolotukhin, and N. Muzyczka Production and purification of recombinant adeno-associated virus Methods Enzymol 316 2000 743 761
28. D. Grimm, A. Kern, M. Pawlita, F. Ferrari, R. Samulski, and J. Kleinschmidt Titration of AAV-2 particles via a novel capsid ELISA: packaging of genomes can limit production of recombinant AAV-2 Gene Ther 6 1999 1322 1330
29. D.K. Rohrer, R. Hartong, and W.H. Dillmann Influence of thyroid hormone and retinoic acid on slow sarcoplasmatic reticulum Ca 2+-ATPase and myosin heavy chain α gene expression in cardiac myocytes J Biol Chem 266 1991 8638 8646
30. R.J. Hajjar, U. Schmidt, T. Matsui, J.L. Guerrero, K.H. Lee, and J.K. Gwathmey Modulation of ventricular function through gene transfer in vivo Proc Natl Acad Sci U S A 95 1998 5251 5256
31. L. Qin, Y. Ding, D.R. Pahud, E. Chang, M.J. Imperiale, and J.S. Bromberg Promoter attenuation in gene therapy: interferon-gamma and tumor necrosis factor-alpha inhibit transgene expression Hum Gene Ther 8 1997 2009 2010
32. A.R. Brooks, R.N. Harkins, P. Wang, H.S. Qian, P. Liu, and G.M. Rubanyi Transcriptional silencing is associated with extensive methylation of the CMV promoter following adenoviral gene delivery to muscle J Gene Med 6 2004 395 404
33. D. Grimm, S. Zhou, H. Nakai, C.E. Thomas, T.A. Storm, and S. Fuess Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy Blood 102 2003 2412 2419
34. A. Handa, S. Muramatsu, J. Qiu, H. Mizukami, and K.E. Brown Adeno-associated virus (AAV)-3-based vectors transduce haematopoietic cells not susceptible to transduction with AAV-2-based vectors J Gen Virol 81 2000 2077 2084
35. K. Pajusola, M. Gruchala, H. Joch, T.F. Luscher, S. Yla-Herttuala, and H. Bueler Cell-type-specific characteristics modulate the transduction efficiency of adeno-associated virus type 2 and restrain infection of endothelial cells J Virol 76 2002 11530 11540
36. K. Hayashi, M. Hayashi, M. Jalkanen, J.H. Firestone, R.L. Trelstad, and M. Bernfield Immunocytochemistry of cell surface heparan sulfate proteoglycan in mouse tissues. A light and electron microscopic study J Histochem Cytochem 35 1987 1079 1088
37. S.J. White, S.A. Nicklin, H. Buning, M.J. Brosnan, K. Leike, and E.D. Papadakis Targeted gene delivery to vascular tissue in vivo by tropism-modified adeno-associated virus vectors Circulation 109 2004 513 519
38. Z. Wang, T. Zhu, C. Qiao, L. Zhou, B. Wang, and J. Zhang Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart Nat Biotechnol 23 2005 321 328
39. L. Du, M. Kido, D.V. Lee, J.E. Rabinowitz, R.J. Samulski, and S.W. Jamieson Differential myocardial gene delivery by recombinant serotype-specific adeno-associated viral vectors Mol Ther 10 2004 604 608
40. P. Gregorevic, M.J. Blankinship, J.M. Allen, R.W. Crawford, L. Meuse, and D.G. Miller Systemic delivery of genes to striated muscles using adeno-associated viral vectors Nat Med 10 2004 828 834
41. O.J. Müller, F. Kaul, M.D. Weitzman, R. Pasqualini, W. Arap, and J.A. Kleinschmidt Random peptide libraries displayed on adeno-associated virus to select for targeted gene therapy vectors Nat Biotechnol 21 2003 1040 1046
42. K.W. Peng, L. Pham, H. Ye, R. Zufferey, D. Trono, and F.L. Cosset Organ distribution of gene expression after intravenous infusion of targeted and untargeted lentiviral vectors Gene Ther 8 2001 1456 1463
43. S.K. Lyons, R. Meuwissen, P. Krimpenfort, and A. Berns The generation of a conditional reporter that enables bioluminescence imaging of Cre/loxP-dependent tumorigenesis in mice Cancer Res 63 2003 7042 7046