Intracoronary Cytoprotective Gene Therapy: A Study of VEGF-B167 in a Pre-Clinical Animal Model of Dilated Cardiomyopathy

Journal of the American College of Cardiology

Volumn 66, Issue 2, 2015, Pages 139-153

  Woitek, Felix ^a,b   Zentilin, Lorena ^c   Hoffman, Nicholas E ^a   Powers, Jeffery C ^a   Ottiger, Isabel ^a,b   Parikh, Suraj ^a   Kulczycki, Anna M ^a   Hurst, Marykathryn ^a   Ring, Nadja ^c   Wang, Tao ^a   Shaikh, Farah ^a   Gross, Polina ^a   Singh, Harinder ^a   Kolpakov, Mikhail A ^a   Linke, Axel ^b   Houser, Steven R ^a   Rizzo, Victor ^a   Sabri, Abdelkarim ^a   Madesh, Muniswamy ^a   Giacca, Mauro ^c   Recchia, Fabio A ^a,d   more..

^a TEMPLE UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b UNIVERSITY OF LEIPZIG   (Germany)

^c INTERNATIONAL CENTRE FOR GENETIC ENGINEERING AND BIOTECHNOLOGY   (Italy)

^d SCUOLA SUPERIORE SANT ANNA   (Italy)

Author keywords

gene therapy; heart failure; translational approach

Indexed keywords

ATRIAL NATRIURETIC FACTOR; GENE THERAPY AGENT; PARVOVIRUS VECTOR; UNCLASSIFIED DRUG; VASCULOTROPIN B167; VASCULOTROPIN B;

ADULT; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CANINE MODEL; CARDIOVASCULAR GENE THERAPY; CELL PROTECTION; CONGESTIVE CARDIOMYOPATHY; CONTROLLED STUDY; DRUG EFFICACY; DRUG SAFETY; HEART PACING; INTRACORONARY DRUG ADMINISTRATION; MALE; NONHUMAN; PRIORITY JOURNAL; PROMOTER REGION; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; ANIMAL; CARDIOMYOPATHY, DILATED; CORONARY BLOOD VESSEL; DISEASE MODEL; DOG; GENE THERAPY; GENETICS; INTRAARTERIAL DRUG ADMINISTRATION; PROCEDURES; TRANSGENE; TRANSLATIONAL RESEARCH; TREATMENT OUTCOME;

ANIMALS; CARDIOMYOPATHY, DILATED; CORONARY VESSELS; DISEASE MODELS, ANIMAL; DOGS; GENETIC THERAPY; INFUSIONS, INTRA-ARTERIAL; MALE; TRANSGENES; TRANSLATIONAL MEDICAL RESEARCH; TREATMENT OUTCOME; VASCULAR ENDOTHELIAL GROWTH FACTOR B;

EID: 84943571489     PISSN: 07351097     EISSN: 15583597     Source Type: Journal    
DOI: 10.1016/j.jacc.2015.04.071     Document Type: Article

References (43)

1. S.T. Pleger, H. Brinks, J. Ritterhoff, and et al. Heart failure gene therapy: the path to clinical practice Circ Res 113 2013 792 809
2. A. Asokan, and R.J. Samulski An emerging adeno-associated viral vector pipeline for cardiac gene therapy Hum Gene Ther 24 2013 906 913
3. S. Zacchigna, L. Zentilin, and M. Giacca Adeno-associated virus vectors as therapeutic and investigational tools in the cardiovascular system Circ Res 114 2014 1827 1846
4. B. Greenberg, A. Yaroshinsky, K.M. Zsebo, and et al. Design of a Phase 2b trial of intracoronary administration of AAV1/SERCA2a in patients with advanced heart failure: the CUPID 2 trial (Calcium Up-Regulation by Percutaneous Administration of Gene Therapy in Cardiac Disease Phase 2B) J Am Coll Cardiol HF 2 2014 84 92
5. J.E. Lähteenvuo, M.T. Lähteenvuo, A. Kivelä, and et al. Vascular endothelial growth factor-B induces myocardium-specific angiogenesis and arteriogenesis via vascular endothelial growth factor receptor-1- and neuropilin receptor-1-dependent mechanisms Circulation 119 2009 845 856
6. L. Zentilin, U. Puligadda, V. Lionetti, and et al. Cardiomyocyte VEGFR-1 activation by VEGF-B induces compensatory hypertrophy and preserves cardiac function after myocardial infarction FASEB J 24 2010 1467 1478
7. M. Pepe, M. Mamdani, L. Zentilin, and et al. Intramyocardial VEGF-B167 gene delivery delays the progression towards congestive failure in dogs with pacing-induced dilated cardiomyopathy Circ Res 106 2010 1893 1903
8. M. Bry, R. Kivelä, V.M. Leppänen, and K. Alitalo Vascular endothelial growth factor-B in physiology and disease Physiol Rev 94 2014 779 794
9. Y. Li, F. Zhang, N. Nagai, and et al. VEGF-B inhibits apoptosis via VEGFR-1-mediated suppression of the expression of BH3-only protein genes in mice and rats J Clin Invest 118 2008 913 923
10. J. Narula, N. Haider, R. Virmani, and et al. Apoptosis in myocytes in end-stage heart failure N Engl J Med 335 1996 1182 1189
11. G. Olivetti, R. Abbi, F. Quaini, and et al. Apoptosis in the failing human heart N Engl J Med 336 1997 1131 1141
12. A. Saraste, K. Pulkki, M. Kallajoki, and et al. Cardiomyocyte apoptosis and progression of heart failure to transplantation Eur J Clin Invest 29 1999 380 386
13. M.J. Everly Cardiac transplantation in the United States: an analysis of the UNOS registry Clin Transpl 2008 35 43
14. B. Olofsson, M. Jeltsch, U. Eriksson, and K. Alitalo Current biology of VEGF-B and VEGF-C Curr Opin Biotechnol 10 1999 528 535
15. D. Cesselli, I. Jakoniuk, L. Barlucchi, and et al. Oxidative stress-mediated cardiac cell death is a major determinant of ventricular dysfunction and failure in dog dilated cardiomyopathy Circ Res 89 2001 279 286
16. D. Abraham, R. Hofbauer, R. Schäfer, and et al. Selective downregulation of VEGFA (165), VEGF-R(1), and decreased capillary density in patients with dilative but not ischemic cardiomyopathy Circ Res 87 2000 644 647
17. F.A. Recchia, P.I. McConnell, R.D. Bernstein, T.R. Vogel, X. Xu, and T.H. Hintze Reduced nitric oxide production and altered myocardial metabolism during the decompensation of pacing-induced heart failure in the conscious dog Circ Res 83 1998 969 979
18. K. Qanud, M. Mamdani, M. Pepe, and et al. Reverse changes in cardiac substrate oxidation in dogs recovering from heart failure Am J Physiol 295 2008 H2098 H2105
19. R.J. Vagnozzi, G.J. Gatto Jr., L.S. Kallander, and et al. Inhibition of the cardiomyocyte-specific kinase TNNI3K limits oxidative stress, injury, and adverse remodeling in the ischemic heart Sci Transl Med 5 2013 207ra141
20. K. Rafiq, M.A. Kolpakov, R. Seqqat, and et al. c-Cbl inhibition improves cardiac function and survival in response to myocardial ischemia Circulation 129 2014 2031 2043
21. B.J. Hawkins, M.D. Levin, P.J. Doonan, and et al. Mitochondrial complex II prevents hypoxic but not calcium- and proapoptotic Bcl-2 protein-induced mitochondrial membrane potential loss J Biol Chem 285 2010 26494 26505
22. B. Yang, and V. Rizzo TNF-alpha potentiates protein-tyrosine nitration through activation of NADPH oxidase and eNOS localized in membrane rafts and caveolae of bovine aortic endothelial cells Am J Physiol Heart Circ Physiol 292 2007 H954 H962
23. R. von Harsdorf, J.G. Edwards, Y.T. Shen, and et al. Identification of a cis-acting regulatory element conferring inducibility of the atrial natriuretic factor gene in acute pressure overload J Clin Invest 100 1997 1294 1304
24. J.G. Edwards In vivo β-adrenergic activation of atrial natriuretic factor (ANF) reporter expression Mol Cell Biochem 29 2006 119 129
25. A. Feldman, P. Ray, C. Silan, J. Mercer, W. Minobe, and M. Bristow Selective gene expression in failing human heart: quantification of steady-state levels of messenger RNA in endomyocardial biopsies using the polymerase chain reaction Circulation 83 1991 1866 1872
26. A. Luchner, D.D. Borgeson, J.A. Grantham, and et al. Relationship between left ventricular wall stress and ANP gene expression during the evolution of rapid ventricular pacing-induced heart failure in the dog Eur J Heart Fail 2 2000 379 386
27. C. Morisco, D.C. Zebrowski, D.E. Vatner, S.F. Vatner, and J. Sadoshima Beta-adrenergic cardiac hypertrophy is mediated primarily by the beta(1)-subtype in the rat heart J Mol Cell Cardiol 33 2001 561 573
28. F.G. Spinale, H.H. Holzgrefe, R. Mukherjee, and et al. LV and myocyte structure and function after early recovery from tachycardia-induced cardiomyopathy Am J Physiol 268 1995 H836 H847
29. J.A. Dixon, and F.G. Spinale Large animal models of heart failure: a critical link in the translation of basic science to clinical practice Circ Heart Fail 2 2009 262 271
30. V. Lionetti, L. Guiducci, A. Simioniuc, and et al. Mismatch between uniform increase in cardiac glucose uptake and regional contractile dysfunction in pacing-induced heart failure Am J Physiol 293 2007 H2747 H2756
31. D. Zablocki, and J. Sadoshima Angiotensin II and oxidative stress in the failing heart Antioxid Redox Signal 19 2013 1095 1109
32. N. Kaludercic, E. Takimoto, T. Nagayama, and et al. Monoamine oxidase A-mediated enhanced catabolism of norepinephrine contributes to adverse remodeling and pump failure in hearts with pressure overload Circ Res 106 2010 193 202
33. M. Meyer, M. Clauss, A. Lepple-Wienhues, and et al. A novel vascular endothelial growth factor encoded by Orf virus, VEGF-E, mediates angiogenesis via signalling through VEGFR-2 (KDR) but not VEGFR-1 (Flt-1) receptor tyrosine kinases EMBO J 18 1999 363 374
34. W. Rottbauer, S. Just, G. Wessels, and et al. VEGF-PLCgamma1 pathway controls cardiac contractility in the embryonic heart Genes Dev 19 2005 1624 1634
35. C.A. Pacak, C.S. Mah, B.D. Thattaliyath, and et al. Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivo Circ Res 99 2006 e3 e9
36. T. Karpanen, M. Bry, H.M. Ollila, T. Seppänen-Laakso, and et al. Overexpression of vascular endothelial growth factor-B in mouse heart alters cardiac lipid metabolism and induces myocardial hypertrophy Circ Res 103 2008 1018 1026
37. C.E. Hagberg, A. Mehlem, A. Falkevall, and et al. Targeting VEGF-B as a novel treatment for insulin resistance and type 2 diabetes Nature 490 2012 426 430
38. H. Su, S. Joho, Y. Huang, and et al. Adeno-associated viral vector delivers cardiac-specific and hypoxia-inducible VEGF expression in ischemic mouse hearts Proc Natl Acad Sci U S A 101 2004 16280 16305
39. A.S. Pachori, L.G. Melo, M.L. Hart, and et al. Hypoxia-regulated therapeutic gene as a preemptive treatment strategy against ischemia/reperfusion tissue injury Proc Natl Acad Sci U S A 101 2004 12282 12307
40. Z. Mallat, I. Philip, M. Lebret, D. Chatel, J. Maclouf, and A. Tedgui Elevated levels of 8-iso-prostaglandin F2alpha in pericardial fluid of patients with heart failure: a potential role for in vivo oxidant stress in ventricular dilatation and progression to heart failure Circulation 97 1998 1536 1539
41. C. Maack, T. Kartes, H. Kilter, and et al. Oxygen free radical release in human failing myocardium is associated with increased activity of rac1-GTPase and represents a target for statin treatment Circulation 108 2003 1567 1574
42. C. Heymes, J.K. Bendall, P. Ratajczak, and et al. Increased myocardial NADPH oxidase activity in human heart failure J Am Coll Cardiol 41 2003 2164 2171
43. M. Satoh, C.M. Matter, H. Ogita, and et al. Inhibition of apoptosis-regulated signaling kinase-1 and prevention of congestive heart failure by estrogen Circulation 115 2007 3197 3204