X-linked inhibitor of apoptosis protein-mediated attenuation of apoptosis, using a novel cardiac-enhanced adeno-associated viral vector

Human Gene Therapy

Volumn 23, Issue 6, 2012, Pages 635-646

  Piacentino, Valentino ^a   Milano, Carmelo A ^a   Bolanos, Michael ^a   Schroder, Jacob ^a   Messina, Emily ^a   Cockrell, Adam S ^b   Jones, Edward ^a   Krol, Ava ^c   Bursac, Nenad ^c   Mao, Lan ^a   Devi, Gayathri R ^a   Jude Samulski, R ^d   Bowles, Dawn E ^a  

^a DUKE UNIVERSITY MEDICAL CENTER   (United States)

^b UNIVERSITY OF NORTH CAROLINA   (United States)

^c Duke University   (United States)

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

Author keywords

[No Author keywords available]

Indexed keywords

BETA ADRENERGIC RECEPTOR STIMULATING AGENT; CASPASE 3; CASPASE 7; PARVOVIRUS VECTOR; PROTEIN KINASE C GAMMA; X LINKED INHIBITOR OF APOPTOSIS;

ANIMAL CELL; ANIMAL CELL CULTURE; ANIMAL EXPERIMENT; ANIMAL MODEL; APOPTOSIS; ARTICLE; CELL DAMAGE; CONTROLLED STUDY; DRUG POTENCY; DRUG TARGETING; ENZYME INHIBITION; GENETIC TRANSDUCTION; HEART FAILURE; HEART LEFT VENTRICLE; HEART MUSCLE CELL; HEART PROTECTION; HEART STRESS; HUMAN; HUMAN TISSUE; HYPOXIA; MALE; MOUSE; NEWBORN; NONHUMAN; PROTEIN EXPRESSION; RAT; TRANSGENE; VIRAL GENE DELIVERY SYSTEM;

ANIMALS; APOPTOSIS; DEPENDOVIRUS; GENETIC VECTORS; HEART FAILURE; HUMANS; MICE; MYOCYTES, CARDIAC; RATS; X-LINKED INHIBITOR OF APOPTOSIS PROTEIN;

RATTUS;

EID: 84864375243     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2011.186     Document Type: Article

References (61)

1. Abbate, A., Biondi-Zoccai, G.G., Bussani, R., et al. (2003). Increased myocardial apoptosis in patients with unfavorable left ventricular remodeling and early symptomatic post-infarction heart failure. J. Am. Coll. Cardiol. 41, 753-760. (Pubitemid 36308381)
2. Bish, L.T., Morine, K., Sleeper, M.M., et al. (2008a). Adenoassociated virus (AAV) serotype 9 provides global cardiac gene transfer superior to AAV1, AAV6, AAV7, and AAV8 in the mouse and rat. Hum. Gene Ther. 19, 1359-1368.
3. Bish, L.T., Sleeper, M.M., Brainard, B., et al. (2008b). Percutaneous transendocardial delivery of self-complementary adeno-associated virus 6 achieves global cardiac gene transfer in canines. Mol. Ther. 16, 1953-1959.
4. Bowles, D.E., McPhee, S.W.J., Li, C., et al. (2011). Phase 1 gene therapy for Duchenne muscular dystrophy using a designer AAV vector. Mol. Ther. 20, 443-455.
5. Chao, H., Liu, Y., Rabinowitz, J., et al. (2000). Several log increase in therapeutic transgene delivery by distinct adeno-associated viral serotype vectors. Mol. Ther. 2, 619-623.
6. Chao, H., Monahan, P.E., Liu, Y., et al. (2001). Sustained and complete phenotype correction of hemophilia B mice following intramuscular injection of AAV1 serotype vectors. Mol. Ther. 4, 217-222. (Pubitemid 32851285)
7. Chatterjee, S., Stewart, A.S., Bish, L.T., et al. (2002). Viral gene transfer of the antiapoptotic factor Bcl-2 protects against chronic postischemic heart failure. Circulation 106, I212-217.
8. Cheng, W., Li, B., Kajstura, J., et al. (1995). Stretch-induced programmed myocyte cell death. J. Clin. Invest. 96, 2247-2259.
9. Czerski, L., and Nunez, G. (2004). Apoptosome formation and caspase activation: Is it different in the heart? J. Mol. Cell. Cardiol. 37, 643-652. (Pubitemid 39194556)
10. Du, L., Kido, M., Lee, D.V., et al. (2004). Differential myocardial gene delivery by recombinant serotype-specific adenoassociated viral vectors. Mol. Ther. 10, 604-608. (Pubitemid 39186121)
11. Gao, G., Bish, L.T., Sleeper, M.M., et al. (2011). Transendocardial delivery of AAV6 results in highly efficient and global cardiac gene transfer in rhesus macaques. Hum. Gene Ther. 22, 979-984.
12. Grieger, J.C., Choi, V.W., and Samulski, R.J. (2006). Production and characterization of adeno-associated viral vectors. Nat. Protoc. 1, 1412-1428.
13. Gustafsson, A.B., and Gottlieb, R.A. (2008). Heart mitochondria: Gates of life and death. Cardiovasc. Res. 77, 334-343. (Pubitemid 351197632)
14. Gwathmey, J.K., Yerevanian, A.I., and Hajjar, R.J. (2011). Cardiac gene therapy with SERCA2a: From bench to bedside. J. Mol. Cell. Cardiol. 50, 803-812.
15. Haider, N., Arbustini, E., Gupta, S., et al. (2009). Concurrent upregulation of endogenous proapoptotic and antiapoptotic factors in failing human hearts. Nat. Clin. Pract. Cardiovasc. Med. 6, 250-261.
16. Hajjar, R.J., Zsebo, K., Deckelbaum, L., et al. (2008). Design of a phase 1/2 trial of intracoronary administration of AAV1/SERCA2a in patients with heart failure. J. Card. Fail. 14, 355-367. (Pubitemid 351756538)
17. Jaski, B.E., Jessup, M.L., Mancini, D.M., et al. (2009). Calcium upregulation by percutaneous administration of gene therapy in cardiac disease (CUPID Trial), a first-in-human phase 1/2 clinical trial. J. Card. Fail. 15, 171-181.
18. Kaiser, J. (2011). Gene therapists celebrate a decade of progress. Science 334, 29-30.
19. Kajstura, J., Cigola, E., Malhotra, A., et al. (1997). Angiotensin II induces apoptosis of adult ventricular myocytes in vitro. J. Mol. Cell. Cardiol. 29, 859-870. (Pubitemid 27196513)
20. Kawamoto, S., Shi, Q., Nitta, Y., et al. (2005). Widespread and early myocardial gene expression by adeno-associated virus vector type 6 with a b-actin hybrid promoter. Mol. Ther. 11, 980-985. (Pubitemid 40724117)
21. Khoynezhad, A., Jalali, Z., and Tortolani, A.J. (2007). A synopsis of research in cardiac apoptosis and its application to congestive heart failure. Tex. Heart Inst. J. 34, 352-359. (Pubitemid 350057579)
22. Kratlian, R.G., and Hajjar, R.J. (2012). Cardiac gene therapy: From concept to reality. Curr. Heart Fail. Rep. 9, 33-39.
23. Laugwitz, K.L., Moretti, A., Weig, H.J., et al. (2001). Blocking caspase-activated apoptosis improves contractility in failing myocardium. Hum. Gene Ther. 12, 2051-2063. (Pubitemid 34053490)
24. Louboutin, J.P., Wang, L., and Wilson, J.M. (2005). Gene transfer into skeletal muscle using novel AAV serotypes. J. Gene Med. 7, 442-451. (Pubitemid 40553125)
25. Masri, C., and Chandrashekhar, Y. (2008). Apoptosis: A potentially reversible, meta-stable state of the heart. Heart Fail. Rev. 13, 175-179.
26. Mingozzi, F., and High, K.A. (2011). Therapeutic in vivo gene transfer for genetic disease using AAV: Progress and challenges. Nat. Rev. Genet. 12, 341-355.
27. Moorjani, N., Westaby, S., Narula, J., et al. (2009). Effects of left ventricular volume overload on mitochondrial and deathreceptor-mediated apoptotic pathways in the transition to heart failure. Am. J. Cardiol. 103, 1261-1268.
28. Okada, H., Takemura, G., Kosai, K., et al. (2005). Postinfarction gene therapy against transforming growth factor-b signal modulates infarct tissue dynamics and attenuates left ventricular remodeling and heart failure. Circulation 111, 2430-2437. (Pubitemid 40696130)
29. Okada, H., Takemura, G., Kosai, K., et al. (2009). Combined therapy with cardioprotective cytokine administration and antiapoptotic gene transfer in postinfarction heart failure. Am J. Physiol. Heart Circ. Physiol. 296, H616-H626.
30. O'Riordan, M.X., Bauler, L.D., Scott, F.L., and Duckett, C.S. (2008). Inhibitor of apoptosis proteins in eukaryotic evolution and development: A model of thematic conservation. Dev. Cell 15, 497-508.
31. Pacak, C.A., and Byrne, B.J. (2011). AAV vectors for cardiac gene transfer: Experimental tools and clinical opportunities. Mol. Ther. 19, 1582-1590.
32. Pacak, C.A., Mah, C.S., Thattaliyath, B.D., Conlon, T.J., et al. (2006). Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivo. Circ. Res. 99, e3-e9. (Pubitemid 44255502)
33. Palomeque, J., Chemaly, E.R., Colosi, P., et al. (2007). Efficiency of eight different AAV serotypes in transducing rat myocardium in vivo. Gene Ther. 14, 989-997.
34. Pedrotty, D.M., Klinger, R.Y., Kirkton, R.D., and Bursac, N. (2009). Cardiac fibroblast paracrine factors alter impulse conduction and ion channel expression of neonatal rat cardiomyocytes. Cardiovasc. Res. 83, 688-697.
35. Pulicherla, N., Shen, S., Yadav, S., et al. (2011). Engineering liverdetargeted AAV9 vectors for cardiac and musculoskeletal gene transfer. Mol. Ther. 19, 1070-1078.
36. Rabinowitz, J.E., Rolling, F., Li, C., et al. (2002). 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, 791-801. (Pubitemid 34033377)
37. Regula, K.M., and Kirshenbaum, L.A. (2005). Apoptosis of ventricular myocytes: A means to an end. J. Mol. Cell. Cardiol. 38, 3-13. (Pubitemid 40038648)
38. Roger, V.L., Go, A.S., Lloyd-Jones, D.M., et al. (2012a). Executive summary: Heart disease and stroke statistics-2012 update: A report from the american heart association. Circulation 125, 188-197.
39. Roger, V.L., Go, A.S., Lloyd-Jones, D.M., et al. (2012b). Heart disease and stroke statistics-2012 update: A report from the American Heart Association. Circulation 125, e2-e220.
40. Romeo, F., Li, D., Shi, M., and Mehta, J.L. (2000). Carvedilol prevents epinephrine-induced apoptosis in human coronary artery endothelial cells: Modulation of Fas/Fas ligand and caspase-3 pathway. Cardiovasc. Res. 45, 788-794. (Pubitemid 30047147)
41. Rossig, L., Haendeler, J., Mallat, Z., et al. (2000). Congestive heart failure induces endothelial cell apoptosis: Protective role of carvedilol. J. Am. Coll. Cardiol. 36, 2081-2089.
42. Savon, C., Acosta, B., Valdes, O., et al. (2008). A myocarditis outbreak with fatal cases associated with adenovirus subgenera C among children from Havana City in 2005. J. Clin. Virol. 43, 152-157.
43. Scheubel, R.J., Bartling, B., Simm, A., et al. (2002). Apoptotic pathway activation from mitochondria and death receptors without caspase-3 cleavage in failing human myocardium: Fragile balance of myocyte survival? J. Am. Coll. Cardiol. 39, 481-488. (Pubitemid 34117506)
44. Stilwell, J.L., and Samulski, R.J. (2004). Role of viral vectors and virion shells in cellular gene expression. Mol. Ther. 9, 337-346. (Pubitemid 38404522)
45. Stilwell, J.L., McCarty, D.M., Negishi, A., et al. (2003). Development and characterization of novel empty adenovirus capsids and their impact on cellular gene expression. J. Virol. 77, 12881-12885. (Pubitemid 37430682)
46. Su, H., Huang, Y., Takagawa, J., et al. (2006). AAV serotype-1 mediates early onset of gene expression in mouse hearts and results in better therapeutic effect. Gene Ther. 13, 1495-1502. (Pubitemid 44567147)
47. Su, H., Yeghiazarians, Y., Lee, A., et al. (2008). AAV serotype 1 mediates more efficient gene transfer to pig myocardium than AAV serotype 2 and plasmid. J. Gene Med. 10, 33-41.
48. Tarantal, A.F., and Lee, C.C. (2009). Long-term luciferase expression monitored by bioluminescence imaging after adeno-associated virus-mediated fetal gene delivery in rhesus monkeys (Macaca mulatta). Hum. Gene Ther. 21, 143-148.
49. Wang, Z., Zhu, T., Qiao, C., et al. (2005). Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart. Nat. Biotechnol. 23, 321-328. (Pubitemid 41094420)
50. Wasala, N.B., Shin, J.H., and Duan, D. (2011). The evolution of heart gene delivery vectors. J. Gene Med. 13, 557-565.
51. Wencker, D., Chandra, M., Nguyen, K., et al. (2003). A mechanistic role for cardiac myocyte apoptosis in heart failure. J. Clin. Invest. 111, 1497-1504. (Pubitemid 38063282)
52. Whelan, R.S., Kaplinskiy, V., and Kitsis, R.N. (2010). Cell death in the pathogenesis of heart disease: Mechanisms and significance. Annu. Rev. Physiol. 72, 19-44.
53. Williams, M.L., and Koch, W.J. (2004). Viral-based myocardial gene therapy approaches to alter cardiac function. Annu. Rev. Physiol. 66, 49-75. (Pubitemid 40614439)
54. Wu, Z., Asokan, A., and Samulski, R.J. (2006). Adeno-associated virus serotypes: Vector toolkit for human gene therapy. Mol. Ther. 14, 316-327. (Pubitemid 44184972)
55. Xiao, W., Chirmule, N., Berta, S.C., et al. (1999). Gene therapy vectors based on adeno-associated virus type 1. J. Virol. 73, 3994-4003. (Pubitemid 29189844)
56. Yamamoto, S., Seta, K., Morisco, C., et al. (2001). Chelerythrine rapidly induces apoptosis through generation of reactive oxygen species in cardiac myocytes. J. Mol. Cell. Cardiol. 33, 1829-1848. (Pubitemid 32977136)
57. Yang, L., Jiang, J., Drouin, L.M., et al. (2009). A myocardium tropic adeno-associated virus (AAV) evolved by DNA shuffling and in vivo selection. Proc. Natl. Acad. Sci. U.S.A. 106, 3946-3951.
58. Yankey, G.K., Li, T., Kilic, A., et al. (2008). Regional remodeling strain and its association with myocardial apoptosis after myocardial infarction in an ovine model. J. Thorac. Cardiovasc. Surg. 135, 991-998, e991-e992.
59. Zincarelli, C., Soltys, S., Rengo, G., and Rabinowitz, J.E. (2008). Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection. Mol. Ther. 16, 1073-1080. (Pubitemid 351737072)
60. Zolotukhin, S., Byrne, B.J., Mason, E., et al. (1999). Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield. Gene Ther. 6, 973-985. (Pubitemid 29295202)
61. Zorc-Pleskovic, R., Alibegovic, A., Zorc, M., et al. (2006). Apoptosis of cardiomyocytes in myocarditis. Folia Biol. (Praha) 52, 6-9. (Pubitemid 44177816)