Gene therapy in the treatment of heart failure

Physiology

Volumn 22, Issue 2, 2007, Pages 81-96

  Ly, Hung ^a   Kawase, Yoshiaki ^a   Yoneyama, Ryuichi ^a   Hajjar, Roger J ^a  

^a MASSACHUSETTS GENERAL HOSPITAL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CALCIUM;

ADENOVIRUS; APOPTOSIS; FORWARD HEART FAILURE; GENE THERAPY; GENE TRANSFER; GENETICS; HEART MUSCLE CELL; HEART MUSCLE CONDUCTION SYSTEM; HUMAN; LENTIVIRINAE; METHODOLOGY; PATHOPHYSIOLOGY; PHYSIOLOGY; REVIEW; TRANSLUMINAL CORONARY ANGIOPLASTY;

ADENOVIRIDAE; ANGIOPLASTY, TRANSLUMINAL, PERCUTANEOUS CORONARY; APOPTOSIS; CALCIUM; CARDIAC OUTPUT, LOW; GENE THERAPY; GENE TRANSFER TECHNIQUES; HEART CONDUCTION SYSTEM; HUMANS; LENTIVIRUS; MYOCYTES, CARDIAC;

EID: 34247245718     PISSN: 15489213     EISSN: 15489221     Source Type: Journal    
DOI: 10.1152/physiol.00037.2006     Document Type: Review

References (86)

1. Barr E, Carroll J, Kalynych AM, Tripathy SK, Kozarsky K, Wilson JM, Leiden JM. Efficient catheter-mediated gene transfer into the heart using replication-defective adenovirus. Gene Ther 1: 51-58, 1994.
2. Beeri R, Guerrero JL, Supple G, Sullivan S, Levine RA, Hajjar RJ. A novel efficient percutaneous myocardial gene delivery system. J Am Coll Cardiol 39: 1181, 2002.
3. Boekstegers P, von Degenfeld G, Giehrl W, Heinrich D, Hullin R, Kupatt C, Steinbeck G, Baretton G, Middeler G, Katus H, Franz WM. Myocardial gene transfer by selective pressure-regulated retroinfusion of coronary veins. Gene Ther 7: 232-240, 2000.
4. Bonci D, Cittadini A, Latronico MV, Borello U, Aycock JK, Drusco A, Innocenzi A, Follenzi A, Lavitrano M, Monti MG, Ross J Jr, Naldini L, Peschle C, Cossu G, Condorelli G. 'Advanced' generation lentiviruses as efficient vectors for cardiomyocyte gene transduction in vitro and in vivo. Gene Ther 10: 630-636, 2003.
5. Braz JC, Bueno OF, Liang Q, Wilkins BJ, Dai YS, Parsons S, Braunwart J, Glascock BJ, Klevitsky R, Kimball TF, Hewett TE, Molkentin JD. Targeted inhibition of p38 MAPK promotes hypertrophic cardiomyopathy through upregulation of calcineurin-NFAT signaling. J Clin Invest 111: 1475-1486, 2003.
6. Braz JC, Gregory K, Pathak A, Zhao W, Sahin B, Klevitsky R, Kimball TF, Lorenz JN, Nairn AC, Liggett SB, Bodi I, Wang S, Schwartz A, Lakatta EG, DePaoli-Roach AA, Robbins J, Hewett TE, Bibb JA, Westfall MV, Kranias EG, Molkentin JD. PKC-alpha regulates cardiac contractility and propensity toward heart failure. Nat Med 10: 248-254, 2004.
7. Bristow MR. beta-adrenergic receptor blockade in chronic heart failure. Circulation 101: 558-569, 2000.
8. Bristow MR. The beta-adrenergic receptor. Configuration, regulation, mechanism of action. Postgrad Med Spec No 19-26: 1988.
9. Bristow MR, Ginsburg R. Beta 2 receptors on myocardial cells in human ventricular myocardium. Am J Cardiol 57: 3F-6F, 1986.
10. Bristow MR, Ginsburg R, Minobe W, Cubicciotti RS, Sageman WS, Lurie K, Billingham ME, Harrison DC, Stinson EB. Decreased catecholamine sensitivity and beta-adrenergic-receptor density in failing human hearts. N Engl J Med 307: 205-211, 1982.
11. Carr AN, Schmidt AG, Suzuki Y, del Monte F, Sato Y, Lanner C, Breeden K, Jing SL, Allen PB, Greengard P, Yatani A, Hoit BD, Grupp IL, Hajjar RJ, DePaoli-Roach AA, Kranias EG. Type 1 phosphatase, a negative regulator of cardiac function. Mol Cell Biol 22: 4124-4135, 2002.
12. Carter PJ, Samulski RJ. Adeno-associated viral vectors as gene delivery vehicles. Int J Mol Med 6: 17-27, 2000.
13. Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G, Gross F, Yvon E, Nusbaum P, Selz F, Hue C, Certain S, Casanova JL, Bousso P, Deist FL, Fischer A. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 288: 669-672, 2000.
14. Champion HC, Georgakopoulos D, Haldar S, Wang L, Wang Y, Kass DA. Robust adenoviral and adeno-associated viral gene transfer to the in vivo murine heart: application to study of phospholamban physiology. Circulation 108: 2790-2797, 2003.
15. Choi VW, Samulski RJ, McCarty DM. Effects of adeno-associated virus DNA hairpin structure on recombination. J Virol 79: 6801-6807, 2005.
16. Christensen G, Minamisawa S, Gruber PJ, Wang Y, Chien KR. High-efficiency, long-term cardiac expression of foreign genes in living mouse embryos and neonates. Circulation 101: 178-184, 2000.
17. Communal C, Huq F, Lebeche D, Mestel C, Gwathmey JK, Hajjar RJ. Decreased efficiency of adenovirus-mediated gene transfer in aging cardiomyocytes. Circulation 107: 1170-1175, 2003.
18. Davia K, Bernobich E, Ranu HK, del Monte F, Terracciano CM, MacLeod KT, Adamson DL, Chaudhri B, Hajjar RJ, Harding SE. SERCA2A overexpression decreases the incidence of aftercontractions in adult rabbit ventricular myocytes. J Mol Cell Cardiol 33: 1005-1015, 2001.
19. del Monte F, Harding SE, Schmidt U, Matsui T, Kang ZB, Dec GW, Gwathmey JK, Rosenzweig A, Hajjar RJ. Restoration of contractile function in isolated cardiomyocytes from failing human hearts by gene transfer of SERCA2a. Circulation 100: 2308-2311, 1999.
20. del Monte F, Williams E, Lebeche D, Schmidt U, Rosenzweig A, Gwathmey JK, Lewandowski ED, Hajjar RJ. Improvement in survival and cardiac metabolism after gene transfer of sarcoplasmic reticulum Ca(2+)-ATPase in a rat model of heart failure. Circulation 104: 1424-1429, 2001.
21. del Monte F, Williams E, Lebeche D, Schmidt U, Rosenzweig A, Gwathmey JK, Lewandowski ED, Hajjar RJ. Improvement in survival and cardiac metabolism after gene transfer of sarcoplasmic reticulum Ca(2+)-ATPase in a rat model of heart failure. Circulation 104: 1424-1429, 2001.
22. Donahue JK, Heldman AW, Fraser H, McDonald AD, Miller JM, Rade JJ, Eschenhagen T, Marban E. Focal modification of electrical conduction in the heart by viral gene transfer. Nat Med 6: 1395-1398, 2000.
23. Donahue JK, Kikkawa K, Johns DC, Marban E, JL. Ultrarapid highly efficient viral gene transfer to the heart. Proc Natl Acad Sci USA 94: 4664-4668, 1997.
24. Donahue JK, Kikkawa K, Thomas AD, Marban E, Lawrence JH. Acceleration of widespread adenoviral gene transfer to intact rabbit hearts by coronary perfusion with low calcium and serotonin. Gene Ther 5: 630-634, 1998.
25. Donato R. S100: a multigenic family of calcium-modulated proteins of the EF-hand type with intracellular and extracellular functional roles. Int J Biochem Cell Biol 33: 637-668, 2001.
26. Edelberg JM, Huang DT, Josephson ME, Rosenberg RD. Molecular enhancement of porcine cardiac chronotropy. Heart 86: 559-562, 2001.
27. El-Armouche A, Pamminger T, Ditz D, Zolk O, Eschenhagen T. Decreased protein and phosphorylation level of the protein phosphatase inhibitor-1 in failing human hearts. Cardiovasc Res 61: 87-93, 2004.
28. Engelhardt S, Hein L, Dyachenkow V, Kranias EG, Isenberg G, Lohse MJ. Altered calcium handling is critically involved in the cardiotoxic effects of chronic beta-adrenergic stimulation. Circulation 109: 1154-1160, 2004.
29. Feldman DS, Carnes CA, Abraham WT, Bristow MR. Mechanisms of disease: beta-adrenergic receptors - alterations in signal transduction and pharmacogenomics in heart failure. Nat Clin Pract Cardiovasc Med 2: 475-483, 2005.
30. Felgner PL. Nonviral strategies for gene therapy. Sci Am 276: 102-106, 1997.
31. Fromes Y, Salmon A, Wang X, Collin H, Rouche A, Hagege A, Schwartz K, Fiszman MY. Gene delivery to the myocardium by intrapericardial injection. Gene Ther 6: 683-688, 1999.
32. Fu H, Muenzer J, Samulski RJ, Breese G, Sifford J, Zeng X, McCarty DM. Self-complementary adeno-associated virus serotype 2 vector: global distribution and broad dispersion of AAV-mediated transgene expression in mouse brain. Mol Ther 8: 911-917, 2003.
33. Galimi F, Noll M, Kanazawa Y, Lax T, Chen C, Grompe M, Verma IM. Gene therapy of Fanconi anemia: preclinical efficacy using lentiviral vectors. Blood 100: 2732-2736, 2002.
34. Galimi F, Verma IM. Opportunities for the use of lentiviral vectors in human gene therapy. Curr Top Microbiol Immunol 261: 245-254, 2002.
35. Guzman RJ, Lemarchand P, Crystal RG, Epstein SE, Finkel T. Efficient and selective adenovirus-mediated gene transfer into vascular neointima. Circulation 88: 2838-2848, 1993.
36. Hacein-Bey-Abina S, Von Kalle C, Schmidt M, McCormack MP, Wulffraat N, Leboulch P, Lim A, Osborne CS, Pawliuk R, Morillon E, Sorensen R, Forster A, Fraser P, Cohen JI, de Saint Basile G, Alexander I, Wintergerst U, Frebourg T, Aurias A, Stoppa-Lyonnet D, Romana S, Radford-Weiss I, Gross F, Valensi F, Delabesse E, Macintyre E, Sigaux F, Soulier J, Leiva LE, Wissler M, Prinz C, Rabbitts TH, Le Deist F, Fischer A, Cavazzana-Calvo M. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302: 415-419, 2003.
37. Hajjar RJ, del Monte F, Matsui T, Rosenzweig A. Prospects for gene therapy for heart failure. Circ Res 86: 616-621, 2000.
38. Hajjar RJ, Schmidt U, Matsui T, Guerrero JL, Lee KH, Gwathmey JK, Dec GW, Semigran MJ, Rosenzweig A. Modulation of ventricular function through gene transfer in vivo. Proc Natl Acad Sci USA 95: 5251-5256, 1998.
39. Hayase M, Del Monte F, Kawase Y, Macneill BD, McGregor J, Yoneyama R, Hoshino K, Tsuji T, De Grand AM, Gwathmey JK, Frangioni JV, Hajjar RJ. Catheter-based antegrade intracoronary viral gene delivery with coronary venous blockade. Am J Physiol Heart Circ Physiol 288: H2995-H3000, 2005.
40. Hoshijima M, Ikeda Y, Iwanaga Y, Minamisawa S, Date MO, Gu Y, Iwatate M, Li M, Wang L, Wilson JM, Wang Y, Ross J Jr, Chien KR. Chronic suppression of heart-failure progression by a pseudophosphorylated mutant of phospholamban via in vivo cardiac rAAV gene delivery. Nat Med 8: 864-871, 2002.
41. Inagaki K, Fuess S, Storm TA, Gibson GA, McTiernan CF, Kay MA, Nakai H. Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8. Mol Ther 14: 45-53, 2006.
42. Isner JM. Myocardial gene therapy. Nature 415: 234-239, 2002.
43. Isner JM, Vale PR, Symes JF, Losordo DW. Assessment of risks associated with cardiovascular gene therapy in human subjects. Circ Res 89: 389-400, 2001.
44. Iwanaga Y, Hoshijima M, Gu Y, Iwatate M, Dieterle T, Ikeda Y, Date MO, Chrast J, Matsuzaki M, Peterson KL, Chien KR, Ross J Jr. Chronic phospholamban inhibition prevents progressive cardiac dysfunction and pathological remodeling after infarction in rats. J Clin Invest 113: 727-736, 2004.
45. Iwatate M, Gu Y, Dieterle T, Iwanaga Y, Peterson KL, Hoshijima M, Chien KR, Ross J. In vivo high-efficiency transcoronary gene delivery and Cre-LoxP gene switching in the adult mouse heart. Gene Ther 10: 1814-1820, 2003.
46. Kaneda Y. Development of a novel fusogenic viral liposome system (HVJ-liposomes) and its applications to the treatment of acquired diseases. Mol Membr Biol 16: 119-122, 1999.
47. Kawaguchi H, Shin WS, Wang Y, Inukai M, Kato M, Matsuo-Okai Y, Sakamoto A, Uehara Y, Kaneda Y, Toyo-oka T. In vivo gene transfection of human endothelial cell nitric oxide synthase in cardiomyocytes causes apoptosis-like cell death Identification using Sendai virus-coated liposomes. Circulation 95: 2441-2447, 1997.
48. Kay MA, Glorioso JC, Naldini L. Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics. Nat Med 7: 33-40, 2001.
49. Klages N, Zufferey R, Trono D. A stable system for the high-titer production of multiply attenuated lentiviral vectors. Mol Ther 2: 170-176, 2000.
50. Koransky ML, Robbins RC, Blau HM. VEGF gene delivery for treatment of ischemic cardiovascular disease. Trends Cardiovasc Med 12: 108-114, 2002.
51. Lathi KG, Vale PR, Losordo DW, Cespedes RM, Symes JF, Esakof DD, Maysky M, Isner JM. Gene therapy with vascular endothelial growth factor for inoperable coronary artery disease: anesthetic management and results. Anesth Analg 92: 19-25, 2001.
52. Mann DL, Kent RL, Parsons B, Cooper Gt. Adrenergic effects on the biology of the adult mammalian cardiocyte. Circulation 85: 790-804, 1992.
53. Manno CS, Pierce GF, Arruda VR, Glader B, Ragni M, Rasko JJ, Ozelo MC, Hoots K, Blatt P, Konkle B, Dake M, Kaye R, Razavi M, Zajko A, Zehnder J, Rustagi PK, Nakai H, Chew A, Leonard D, Wright JF, Lessard RR, Sommer JM, Tigges M, Sabatino D, Luk A, Jiang H, Mingozzi F, Couto L, Ertl HC, High KA, Kay MA. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med 12: 342-347, 2006.
54. McCarty DM, Fu H, Monahan PE, Toulson CE, Naik P, Samulski RJ. Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo. Gene Ther 10: 2112-2118, 2003.
55. McCarty DM, Monahan PE, Samulski RJ. Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis. Gene Ther 8: 1248-1254, 2001.
56. Monahan PE, Samulski RJ. AAV vectors: is clinical success on the horizon? Gene Ther 7: 24-30, 2000.
57. Monahan PE, Samulski RJ. Adeno-associated virus vectors for gene therapy: more pros than cons? Mol Med Today 6: 433-440, 2000.
58. Monahan PE, Samulski RJ, Tazelaar J, Xiao X, Nichols TC, Bellinger DA, Read MS, Walsh CE. Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia. Gene Ther 5: 40-49, 1998.
59. Most P, Pleger ST, Völkers M, Heidt B, Boerries M, Weichenhan D, Löffler M, Janssen PML, Eckhart AD, Martini J, Williams ML, Katus H, Remppis A, Koch WJ. Cardiac adenoviral S100A1 gene delivery rescues failing myocardium. J Clin Invest 114: 1550-1563, 2004.
60. Nabel EG. Gene therapy for cardiovascular disease. Circulation 91: 541-548, 1995.
61. Nabel EG. Gene therapy for vascular diseases. Atherosclerosis 118, Suppl: S51-S56, 1995.
62. Nabel EG, Simari R, Yang Z, San H, Nabel GJ. In vivo gene transfer: a biological tool. Ann NY Acad Sci 811: 289-292, 1997.
63. Nabel GJ. Genetic, cellular and immune approaches to disease therapy: past and future. Nat Med 10: 135-141, 2004.
64. Nabel GJ. The future of gene therapy. Ernst Schering Res Found Workshop 43: 1-16, 2003.
65. Nagata K, Marban E, Lawrence JH, Donahue JK. Phosphodiesterase inhibitor-mediated potentiation of adenovirus delivery to myocardium. J Mol Cell Cardiol 33: 575-580, 2001.
66. Nishikawa M, Huang L. Nonviral vectors in the new millennium: delivery barriers in gene transfer. Hum Gene Ther 12: 861-870, 2001.
67. Pacak CA, Mah CS, Thattaliyath BD, Conlon TJ, Lewis MA, Cloutier DE, Zolotukhin I, Tarantal AF, Byrne BJ. Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivo. Circ Res 99: e3-e9, 2006.
68. Pathak A, del Monte F, Zhao W, Schultz JE, Lorenz JN, Bodi I, Weiser D, Hahn H, Carr AN, Syed F, Mavila N, Jha L, Qian J, Marreez Y, Chen G, McGraw DW, Heist EK, Guerrero JL, DePaoli-Roach AA, Hajjar RJ, Kranias EG. Enhancement of cardiac function and suppression of heart failure progression by inhibition of protein phosphatase 1 [see comment]. Circ Res 96: 756-766, 2005.
69. Port JD, Bristow MR. Altered beta-adrenergic receptor gene regulation and signaling in chronic heart failure. J Mol Cell Cardiol 33: 887-905, 2001.
70. Preovolos AC, Mennen MT, Bilney A, Mariani J, Kaye DM, Power JM. Development of a novel perfusion technique to allow targeted delivery of gene therapy: the V-Focus system. J Extra Corpor Technol 38: 51-52, 2006.
71. Qin XQ, Tao N, Dergay A, Moy P, Fawell S, Davis A, Wilson JM, Barsoum J. Interferon-beta gene therapy inhibits tumor formation and causes regression of established tumors in immune-deficient mice. Proc Natl Acad Sci USA 95: 14411-14416, 1998.
72. Santiago FS, Khachigian LM. Nucleic acid based strategies as potential therapeutic tools: mechanistic considerations and implications to restenosis. J Mol Med 79: 695-706, 2001.
73. Sato M, O'Gara P, Harding SE, Fuller SJ. Enhancement of adenoviral gene transfer to adult rat cardiomyocytes in vivo by immobilization and ultrasound treatment of the heart. Gene Ther 12: 936-941, 2005.
74. Shah AS, White DC, Emani S, Kypson AP, Lilly RE, Wilson K, Glower DD, Lefkowitz RJ, Koch WJ. In vivo ventricular gene delivery of a beta-adrenergic receptor kinase inhibitor to the failing heart reverses cardiac dysfunction. Circulation 103: 1311-1316, 2001.
75. Shohet RV, Chen S, Zhou YT, Wang Z, Meidell RS, Unger RH, Grayburn PA. Echocardiographic destruction of albumin microbubbles directs gene delivery to the myocardium. Circulation 101: 2554-2556, 2000.
76. Sirven A, Pflumio F, Zennou V, Titeux M, Vainchenker W, Coulombel L, Dubart-Kupperschmitt A, Charneau P. The human immunodeficiency virus type-1 central DNA flap is a crucial determinant for lentiviral vector nuclear import and gene transduction of human hematopoietic stem cells. Blood 96: 4103-4110, 2000.
77. Terracciano C. Functional consequences of Na/Ca exchanger overexpression in cardiac myocytes. Ann NY Acad Sci 976: 520-527, 2002.
78. Tse HF, Xue T, Lau CP, Siu CW, Wang K, Zhang QY, Tomaselli GF, Akar FG, Li RA. Bioartificial sinus node constructed via in vivo gene transfer of an engineered pacemaker HCN Channel reduces the dependence on electronic pacemaker in a sick-sinus syndrome model. Circulation 114: 1000-1011, 2006.
79. Vale PR, Losordo DW, Milliken CE, McDonald MC, Gravelin LM, Curry CM, Esakof DD, Maysky M, Symes JF, Isner JM. Randomized, single-blind, placebo-controlled pilot study of catheter-based myocardial gene transfer for therapeutic angiogenesis using left ventricular electromechanical mapping in patients with chronic myocardial ischemia. Circulation 103: 2138-2143, 2001.
80. Wahlfors J, Morgan RA. Semliki Forest virus vectors for gene transfer. Methods Mol Med 76: 493-502, 2003.
81. 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.
82. Xiao X, Li J, McCown TJ, Samulski RJ. Gene transfer by adeno-associated virus vectors into the central nervous system. Exp Neurol 144: 113-124, 1997.
83. Xiao X, McCown TJ, Li J, Breese GR, Morrow AL, Samulski RJ. Adeno-associated virus (AAV) vector antisense gene transfer in vivo decreases GABA(A) alpha1 containing receptors and increases inferior collicular seizure sensitivity. Brain Res 756: 76-83, 1997.
84. Yang CC, Xiao X, Zhu X, Ansardi DC, Epstein ND, Frey MR, Matera AG, Samulski RJ. Cellular recombination pathways and viral terminal repeat hairpin structures are sufficient for adeno-associated virus integration in vivo and in vitro. J Virol 71: 9231-9247, 1997.
85. Zhou YY, Lakatta EG, Xiao RP. Age-associated alterations in calcium current and its modulation in cardiac myocytes. Drugs Aging 13: 159-171, 1998.
86. Zhu T, Zhou L, Mori S, Wang Z, McTiernan CF, Qiao C, Chen C, Wang DW, Li J, Xiao X. Sustained whole-body functional rescue in congestive heart failure and muscular dystrophy hamsters by systemic gene transfer. Circulation 112: 2650-2659, 2005.