Gene therapy: Targeting the myocardium

Heart

Volumn 94, Issue 1, 2008, Pages 89-99

  Lyon, Alexander R ^a,b   Sato, M ^a   Hajjar, R J ^b   Samulski, R J ^c   Harding, S E ^a  

^a NATIONAL HEART AND LUNG INSTITUTE   (United Kingdom)

^b MOUNT SINAI HOSPITAL   (United States)

^c UNIVERSITY OF NORTH CAROLINA SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

COXSACKIE VIRUS AND ADENOVIRUS RECEPTOR; DOUBLE STRANDED DNA; DOUBLE STRANDED RNA; FIBROBLAST GROWTH FACTOR 4; HERPES SIMPLEX VIRUS VECTOR; LENTIVIRUS VECTOR; PARVOVIRUS VECTOR; PLASMID DNA; POXVIRUS VECTOR; RETROVIRUS VECTOR; SARCOPLASMIC RETICULUM CALCIUM TRANSPORTING ADENOSINE TRIPHOSPHATASE; SINGLE STRANDED DNA; SINGLE STRANDED RNA; UNCLASSIFIED DRUG; VACCINIA VIRUS VECTOR; VASCULOTROPIN;

ALANINE AMINOTRANSFERASE BLOOD LEVEL; ASPARTATE AMINOTRANSFERASE BLOOD LEVEL; CLINICAL TRIAL; GENE EXPRESSION; GENE STRUCTURE; GENE TARGETING; GENE THERAPY; GENETIC TRANSFECTION; HEART FAILURE; HEART MUSCLE; HEART MUSCLE ISCHEMIA; HUMAN; LONG QT SYNDROME; MYOCARDIAL DISEASE; NONHUMAN; PRIORITY JOURNAL; REVIEW; RNA TRANSLATION; RNA TRANSPORT; SIDE EFFECT; STABLE ANGINA PECTORIS; THROMBOCYTOPENIA; TRANSGENE; UNSPECIFIED SIDE EFFECT; VIRAL GENE DELIVERY SYSTEM; VIROGENESIS; VIRUS CAPSID;

CARDIOMYOPATHIES; DEPENDOVIRUS; GENE EXPRESSION REGULATION, VIRAL; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS; LENTIVIRUS; PRACTICE GUIDELINES AS TOPIC; RETROVIRIDAE;

EID: 37749013444     PISSN: 13556037     EISSN: None     Source Type: Journal    
DOI: 10.1136/hrt.2007.116483     Document Type: Review

References (94)

1. European Society of Gene and Cell Therapy. Position paper on social, ethical and public awareness issues in gene therapy. 2002.
2. Rosenberg SA, Aebersold P, Cornetta K, et al. Gene transfer into humans - immunotherapy of patients with advanced melanoma, using tumor-infiltrating lymphocytes modified by retroviral gene transduction. N Engl J Med 1990;323:570-8.
3. Lehrman S. Virus treatment questioned after gene therapy death. Nature 1999;401:517-8.
4. Check E. Sanctions agreed over teenager's gene-therapy death. Nature 2005;433:674.
5. Hacein-Bey-Abina S, Von Kalle C, Schmidt M, et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 2003;302:415-9.
6. Morgan RA, Dudley ME, Wunderlich JR, et al. Cancer regression in patients after transfer of genetically engineered lymphocytes. Science 2006;314:126-9.
7. Cristofanilli M, Krishnamurthy S, Guerra L, et al. A nonreplicating adenoviral vector that contains the wild-type p53 transgene combined with chemotherapy for primary breast cancer: safety, efficacy, and biologic activity of a novel gene-therapy approach Cancer 2006;107:935-44.
8. Jiang H, Pierce GF, Ozelo MC, et al. Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B. Mol Ther 2006;14:452-5.
9. Sarkar N, Blomberg P, Wardell E, et al. Nonsurgical direct delivery of plasmid DNA into rat heart: time course, dose response, and the influence of different promoters on gene expression. J Cardiovasc Pharmacol 2002;39:215-24.
10. Bauer S, Maier SK, Neyses L, et al. Optimization of gene transfer into neonatal rat cardiomyocytes and unmasking of cytomegalovirus promoter silencing. DNA Cell Biol 2005;24:381-7.
11. Poelzing S, Rosenbaum DS. Nature, significance, and mechanisms of electrical heterogeneities in ventricle. Anat Rec A Discov Mol Cell Evol Biol 2004;280:1010-17.
12. Yue AM, Franz MR, Roberts PR, et al. Global endocardial electrical restitution in human right and left ventricles determined by noncontact mapping. J Am Coll Cardiol 2005;46:1067-75.
13. Choi VW, McCarty DM, Samulski RJ. Host cell DNA repair pathways in adeno-associated viral genome processing. J Virol 2006;80:10346- 56.
14. Bamford KB, Wood S, Shaw RJ. Standards for gene therapy clinical trials based on pro-active risk assessment in a London NHS Teaching Hospital Trust. QJM 2005;98:75-86.
15. Losordo DW, Vale PR, Symes JF, et al. Gene therapy for myocardial angiogenesis: initial clinical results with direct myocardial injection of phVEGF165 as sole therapy for myocardial ischemia. Circulation 1998;98:2800-4.
16. Losordo DW, Vale PR, Hendel RC, et al. Phase 1/2 placebo-controlled, double-blind, dose-escalating trial of myocardial vascular endothelial growth factor 2 gene transfer by catheter delivery in patients with chronic myocardial ischemia. Circulation 2002;105:2012-8.
17. Kastrup J, Jorgensen E, Ruck A, et al. Direct mtramyocardial plasmid vascular endothelial growth factor-A165 gene therapy in patients with stable severe angina pectoris: A randomized double-blind placebo-controlled study: The Euroinject One trial. J Am Coll Cardiol 2005;45:982-8.
18. Wright MJ, Wightman LML, Lilley C, et al. In vivo myocardial gene transfer: Optimization, evaluation and direct comparison of gene transfer vectors. Basic Res Cardiol 2001;96:227-36.
19. Rutanen J, Rissanen TT, Markkanen JE, et al. Adenoviral catheter-mediated intramyocardial gene transfer using the mature form of vascular endothelial growth factor-D induces transmural angiogenesis in porcine heart. Circulation 2004;109:1029-35.
20. Miyagawa S, Sawa Y, Taketani S, et al. Myocardial regeneration therapy for heart failure: hepatocyte growth factor enhances the effect of cellular cardiomyoplasty. Circulation 2002;105:2556-61.
21. Lee M, Rentz J, Han SO, et al. Water-soluble lipopolymer as an efficient carrier for gene delivery to myocardium. Gene Ther 2003;10:585-93.
22. Tokunaga N, Nagaya N, Shirai M, et al. Adrenomedullin gene transfer induces therapeutic angiogenesis in a rabbit model of chronic hind limb ischemia: benefits of a novel nonviral vector, gelatin. Circulation 2004;109:526-31.
23. Bekeredjian R, Chen S, Frenkel PA, et al. Ultrasound-targeted microbubble destruction can repeatedly direct highly specific plasmid expression to the heart. Circulation 2003;108:1022-6.
24. Kondo I, Ohmori K, Oshita A, et al. Treatment of acute myocardial infarction by hepatocyte growth factor gene transfer: the first demonstration of myocardial transfer of a "functional" gene using ultrasonic microbubble destruction. J Am Coll Cardiol 2004;44:644-53.
25. Ogawa K, Tachibana K, Uchida T, et al. High-resolution scanning electron microscopic evaluation of cell-membrane porosity by ultrasound. Med Electron Microsc 2001;34:249-53.
26. Tsunoda S, Mazda O, Oda Y, et al. Sonoporation using microbubble BR14 promotes pDNA/siRNA transduction to murine heart. Biochem Biophys Res Commun 2005;336:118-27.
27. Kim JM, Lim BK, Ho SH, et al. TNFR-Fc fusion protein expressed by in vivo electroporation improves survival rates and myocardial injury in coxsackievirus induced murine myocarditis. Biochem Biophys Res Commun 2006;344:765-71.
28. Iida Y, Oda Y, Nakamori S, et al. Transthoracic direct current shock facilitates intramyocardial transfection of naked plasmid DNA infused via coronary vessels in canines. Gene Ther 2006;13:906-16.
29. Lange A, Mills RE, Lange CJ, et al. Classical nuclear localization signals: Definition, function, and interaction with importin alpha. J Biol Chem 2007;282:5101-5.
30. Munkonge FM, Dean DA, Hillery E, et al. Emerging significance of plasmid DNA nuclear import in gene therapy. Adv Drug Deliv Rev 2003;55:749-60.
31. Shiraishi T, Hamzavi R, Nielsen PE. Targeted delivery of plasmid DNA into the nucleus of cells via nuclear localization signal peptide conjugated to DNA intercalating bis- and trisacridines. Bioconjug Chem 2005;16:1112-6.
32. Rolland A. Nuclear gene delivery: the Trojan horse approach. Expert Opin Drug Deliv 2006;3:1-10.
33. Schirmbeck R, Konig-Merediz SA, Riedl P, et al. Priming of immune responses to hepatitis B surface antigen with minimal DNA expression constructs modified with a nuclear localization signal peptide. J Mol Med 2001;79:343-50.
34. Rota M, Boni A, Urbanek K, et al. Nuclear targeting of Akt enhances ventricular function and myocyte contractility. Circ Res 2005;97:1332-41.
35. Byun J, Huh JE, Park SJ, et al. Myocardial injury-induced fibroblast proliferation facilitates retroviral-mediated gene transfer to the rat heart in vivo. J Gene Med 2000:22-10.
36. Donahue RE, Kessler SW, Bodine D, et al. Helper virus induced T cell lymphoma in nonhuman primates after retroviral mediated gene transfer. J Exp Med 1992;176:1125-35.
37. Zhao J, Pettigrew GJ, Thomas J, et al. Lentiviral vectors for delivery of genes into neonatal and adult ventricular cardiac myocytes in vitro and in vivo. Basic Res Cardiol 2002;97:348-58.
38. Bonci D, Cittadini A, Latronico MV, et al. 'Advanced' generation lentiviruses as efficient vectors for cardiomyocyte gene transduction in vitro and in vivo. Gene Therapy 2003;10:630-6.
39. Yoshimitsu M, Higuchi K, Dawood F, et al. Correction of cardiac abnormalities in fabry mice by direct intraventricular injection of a recombinant lentiviral vector that engineers expression of alpha-galactosidase A. Circ J 2006;70:1503-8.
40. Sakoda T, Kasahara N, Kedes L, et al. Lentiviral vector-mediated gene transfer to endotherial cells compared with adenoviral and retroviral vectors. Prep Biochem Biotechnol 2007;37:1-11.
41. Miyoshi H, Blomer U, Takahashi M, et al. Development of a self-inactivating lentivirus vector. J Virol 1998;72:8150-7.
42. Bahner I, Sumiyoshi T, Kagoda M, et al. Lentiviral Vector Transduction of a Dominant-negative Rev Gene Into Human CD34+ Hematopoietic Progenitor Cells Potently Inhibits Human Immunodeficiency Virus-1 Replication. Mol Ther 2007;15:76-85.
43. Azzouz M, Ralph GS, Storkebaum E, et al. VEGF delivery with retrogradely transported lentivector prolongs survival in a mouse ALS model. Nature 2004;429:413-7.
44. Yanez-Munoz RJ, Balaggan KS, MacNeil A, et al. Effective gene therapy with nonintegrating lentiviral vectors. Nat Med 2006;12:348-53.
45. Guzman RJ, Lemarchand P, Crystal RG, et al. Efficient gene transfer into myocardium by direct injection of adenovirus vectors. Circ Res 1993;73:1202-7.
46. French BA, Mazur W, Geske RS, et al. Direct in vivo gene transfer into porcine myocardium using replication-deficient adenoviral vectors. Circulation 1994;90:2414-24.
47. Volpers C, Kochanek S. Adenoviral vectors for gene transfer and therapy. J Gene Med 2004;6(Suppl 1):S164-S171.
48. Calabrese F, Thiene G. Myocarditis and inflammatory cardiomyopathy: microbiological and molecular biological aspects. Cardiovasc Res 2003;60:11-25.
49. Yang Y, Nunes FA, Berencsi K, et al. Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy. Proc Natl Acad Sci U S A 1994;91:4407-11.
50. Schiedner G, Morral N, Parks RJ, et al. Genomic DNA transfer with a high-capacity adenovirus vector results in improved in vivo gene expression and decreased toxicity. Nat Genet 1998;18:180-3.
51. Chen HH, Mack LM, Kelly R, et al. Persistence in muscle of an adenoviral vector that lacks all viral genes. Proc Natl Acad Sci U S A 1997;94:1645-50.
52. Fleury S, Driscoll R, Simeoni E, et al. Helper-dependent adenovirus vectors devoid of all viral genes cause less myocardial inflammation compared with first-generation adenovirus vectors. Basic Res Cardiol 2004;99:247-56.
53. Wickham TJ, Carrion ME, Kovesdi I. Targeting of adenovirus penton base to new receptors through replacement of its RGD motif with other receptor-specific peptide motifs. Gene Ther 1995;2:750-6.
54. Havenga MJE, Lemckert AAC, Grimbergen JM, et al. Improved adenovirus vectors for infection of cardiovascular tissues. J Virol 2001;75:3335-42.
55. Thirion C, Lochmuller H, Ruzsics Z, et al. Adenovirus vectors based on human adenovirus type 19a have high potential for human muscle-directed gene therapy. Hum Gene Ther 2006;17:193-205.
56. Communal C, Huq F, Lebeche D, et al. Decreased efficiency of adenovirus-mediated gene transfer in aging cardiomyocytes. Circulation 2003;107:1170-5.
57. Vorburger SA, Hunt KK. Adenoviral Gene Therapy. Oncologist 2002;7:46-59.
58. Rissanen TT, Yla-Herttuala S. Current status of cardiovascular gene therapy. Mol Ther 2007;15:1233-47.
59. Grines CL, Watkins MW, Mahmarian JJ, et al. A randomized, double-blind, placebo-controlled trial of Ad5FGF-4 gene therapy and its effect on myocardial perfusion in patients with stable angina. J Am Coll Cardiol 2003;42:1339-47.
60. Grines CL. The AGENT clinical trials programme. Eur Heart J Suppl 2004;6(suppl_E):E18-E23.
61. Stewart DJ, Hilton JD, Arnold JMO, et al. Angiogenic gene therapy in patients with nonrevascularizable ischemic heart disease: a phase 2 randomized, controlled trial of AdVEGF(121) (AdVEGF121) versus maximum medical treatment. Gene Ther 2006;13:1503-11.
62. Palomeque J, Chemaly ER, Colosi P, et al. Efficiency of eight different AAV serotypes in transducing rat myocardium in vivo. Gene Ther 2007;14:989-97.
63. Pacak CA, Mah CS, Thattaliyath BD, et al. Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivo. Circ Res 2006;99:e3-e9.
64. Chu D, Sullivan CC, Weitzman MD, et al. Direct comparison of efficiency and stability of gene transfer into the mammalian heart using adeno-associated virus versus adenovirus vectors. J Thorac Cardiovasc Surg 2003;126:671-9.
65. Svensson EC, Marshall DJ, Woodard K, et al. Efficient and stable transduction of cardiomyocytes after intramyocardial injection or intracoronary perfusion with recombinant adeno-associated virus vectors. Circulation 1999;99:201-5.
66. Ferrarini M, Arsic N, Recchia FA, et al. Adeno-associated virus-mediated transduction of VEGF165 improves cardiac tissue viability and functional recovery after permanent coronary occlusion in conscious dogs. Circ Res 2006;98:954-61.
67. Kaspar BK, Roth DM, Lai NC, et al. Myocardial gene transfer and long-term expression following intracoronary delivery of adeno-associated virus. J Gene Med 2005;7:316-24.
68. Vandendriessche T, Thorrez L, Acosta-Sanchez A, et al. Efficacy and safety of adeno-associated viral vectors based on serotype 8 and 9 vs. lentiviral vectors for hemophilia B gene therapy. J Thromb Haemost 2007;5:16-24.
69. Jooss K, Yang Y, Fisher KJ, et al. Transduction of dendritic cells by DNA viral vectors directs the immune response to transgene products in muscle fibers. J Virol 1998;72:4212-23.
70. Manno CS, Arruda VR, Pierce GF, et al. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med 2006;12:342-7.
71. Di Pasquale G, Chiorini JA. AAV Transcytosis through Barrier Epithelia and Endothelium. Mol Ther 2006;13:506-16.
72. McCarty DM, Young SM Jr, Samulski RJ. Integration of adeno-associated virus (AAV) and recombinant AAV vectors. Annu Rev Genet 2004;38:819-45.
73. Lai Y, Yue Y, Liu M, et al. Efficient in vivo gene expression by trans-splicing adeno-associated viral vectors. Nat Biotechnol 2005;23:1435-9.
74. Ostedgaard LS, Rokhlina T, Karp PH, et al. A shortened adeno-associated virus expression cassette for CFTR gene transfer to cystic fibrosis airway epithelia. Proc Natl Acad Sci U S A 2005;102:2952- 7.
75. McCarty DM, Fu H, Monahan PE, et al. Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo. Gene Ther 2003;10:2112-8.
76. Wang Z, Ma HI, Li J, et al. Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo. Gene Ther 2003;10:2105-11.
77. Erles K, Sebokova P, Schlehofer JR. Update on the prevalence of serum antibodies (IgG and IgM) to adeno-associated virus (AAV). J Med Virol 1999;59:406-11.
78. Peden CS, Burger C, Muzyczka N, et al. Circulating anti-wild-type adeno-associated virus type 2 (AAV2) antibodies inhibit recombinant AAV2 (rAAV2)-mediated, but not rAAV5-mediated, gene transfer in the brain. J Virol 2004;78:6344-59.
79. Rabinowitz JE, Bowles DE, Faust SM, et al. Cross-dressing the virion: the transcapsidation of adeno-associated virus serotypes functionally defines subgroups. J Virol 2004;78:4421-32.
80. Bowles DE, Rabinowitz JE, Samulski RJ. Marker rescue of adeno-associated virus (AAV) capsid mutants: a novel approach for chimeric AAV production. J Virol 2003;77:423-32.
81. Wu Z, Asokan A, Grieger JC, et al. Single amino acid changes can influence titer, heparin binding, and tissue tropism in different adeno-associated virus serotypes. J Virol 2006;80:11393-7.
82. Carter BJ. Adeno-associated virus vectors in clinical trials. Hum Gene Ther 2005;16:541-50.
83. Franz WM, Breves D, Klingel K, et al. Heart-specific targeting of firefly luciferase by the myosin light chain-2 promoter and developmental regulation in transgenic mice. Circ Res 1993;73:629-38.
84. Pachori AS, Melo LG, Hart ML, et al. Hypoxia-regulated therapeutic gene as a preemptive treatment strategy against ischemia/reperfusion tissue injury. Proc Natl Acad Sci U S A 2004;101:12282-7.
85. Loeb JE, Cordier WS, Harris ME, et al. Enhanced expression of transgenes from adeno-associated virus vectors with the woodchuck hepatitis virus posttranscriptional regulatory element: implications for gene therapy. Hum Gene Ther 1999;10:2295-305.
86. Suarez J, Gloss B, Belke DD, et al. Doxycycline inducible expression of SERCA2a improves calcium handling and reverts cardiac dysfunction in pressure overload-induced cardiac hypertrophy. Am J Physiol Heart Circ Physiol 2004;287:H2164-H2172.
87. Rana TM. Illuminating the silence: understanding the structure and function of small RNAs. Nat Rev Mol Cell Biol 2007;8:23-36.
88. del Monte F, Hajjar RJ. Targeting calcium cycling proteins in heart failure through gene transfer. J Physiol (Lond) 2003;546:49-61.
89. Ripa RS, Wang Y, Jorgensen E, et al. Intramyocardial injection of vascular endothelial growth factor-A165 plasmid followed by granulocyte-colony stimulating factor to induce angiogenesis in patients with severe chronic ischaemic heart disease. Eur Heart J 2006;27:1785- 92.
90. Chen S, Shohet RV, Bekeredjian R, et al. Optimization of ultrasound parameters for cardiac gene delivery of adenoviral or plasmid deoxyribonucleic acid by ultrasound-targeted microbubble destruction. J Am Coll Cardiol 2003;42:301-8.
91. Sato M, O'Gara P, Harding SE, et al. Enhancement of adenoviral gene transfer to adult rat cardiomyocytes in vivo by immobilization and ultrasound treatment of the heart. Gene Therapy 2005;12:936- 41.
92. Lippin Y, Dranitzki-Elhalel M, Brill-Almon E, et al. Human erythropoietin gene therapy for patients with chronic renal failure. Blood 2005;106:2280-6.
93. Suzuki N, Nishii K, Okazaki T, et al. Human artificial chromosomes constructed using the bottom-up strategy are stably maintained in mitosis and efficiently transmissible to progeny mice. J Biol Chem 2006;281:26615-23.
94. Gene Therapy Clinical Trials Worldwide (provided by the Journal of Gene Medicine). Available from http://www.wiley.co.uk/genmed/clinical/ (accessed 20 July 2007).