Journal of Molecular and Cellular Cardiology
Volume 50, Issue 5 , Pages 759-765 , May 2011

Gene therapy strategies for cardiac electrical dysfunction

  • Ian Greener
  • ,
  • J. Kevin Donahue

      Affiliations

    • Corresponding Author InformationCorresponding author. Heart and Vascular Research Center, MetroHealth Hospital, Case Western Reserve University School of Medicine, Rammelkamp 653, 2500 MetroHealth Drive, Cleveland, OH 44109, USA. Tel.: +1 216 778 7342; fax: +1 216 778 1261.

Received 3 June 2010 ,Revised 29 July 2010 ,Accepted 30 July 2010.

References 

  1. Fox CS, Evans JC, Larson MG, Kannel WB, Levy D. Temporal trends in coronary heart disease mortality and sudden cardiac death from 1950 to 1999: the Framingham Heart Study. Circulation. 2004;110:522–527
  2. Echt DS, Liebson PR, Mitchell LB, Peters RW, Obias-Manno D, Barker AH, et al. Mortality and morbidity in patients receiving encainide, flecainide, or placebo. The Cardiac Arrhythmia Suppression Trial. N. Engl. J. Med. 1991;324:781–788
  3. Lipskaia L, Chemaly ER, Hadri L, Lompre AM, Hajjar RJ. Sarcoplasmic reticulum Ca(2+) ATPase as a therapeutic target for heart failure. Expert. Opin. Biol. Ther. 2010;10:29–41
  4. Kikuchi K, McDonald AD, Sasano T, Donahue JK. Targeted modification of atrial electrophysiology by homogeneous transmural atrial gene transfer. Circulation. 2005;111:264–270
  5. Sasano T, Kikuchi K, McDonald AD, Lai S, Donahue JK. Targeted high-efficiency, homogeneous myocardial gene transfer. J. Mol. Cell Cardiol. 2007;42:954–961
  6. Nattel S, Maguy A, Le BS, Yeh YH. Arrhythmogenic ion-channel remodeling in the heart: heart failure, myocardial infarction, and atrial fibrillation. Physiol. Rev. 2007;87:425–456
  7. Lin H, Parmacek M, Morle G, Bolling S, Leiden J. Expression of recombinant genes in the myocardium in vivo after direct injection of DNA. Circulation. 1990;82:2217–2221
  8. Shenk T. In: Adenoviridae: the viruses and their replication. Fields of Virology. 3rd ed. Philadelphia: Lippincott-Raven Publishers; 2010;p. 2111–2148
  9. Ferrari FK, Samulski T, Shenk T, Samulski RJ. Second-strand synthesis is a rate-limiting step for efficient transduction by recombinant adeno-associated virus vectors. J. Virol. 1996;70:3227–3234
  10. Hauck B, Zhao W, High K, Xiao W. Intracellular viral processing, not single-stranded DNA accumulation, is crucial for recombinant adeno-associated virus transduction. J. Virol. 2004;78:13678–13686
  11. High K, Tigges M, Manno C, Sabatino D, Arruda V, Herzog R, et al. Human immune responses to AAV-2 capsid may limit duration of expression in liver-directed gene transfer in humans with hemophilia B. Blood. 2004;104:413
  12. Cleghorn FR, Reitz MS, Popovic M, et al. Human immunodeficiency viruses. In:  Mandell GL,  Bennett JE,  Dolin R editor. Principles and Practice of Infectious Diseases. 2010;p. 2119–2130
  13. Bonci D, Cittadini A, Latronico MV, Borello U, Aycock JK, Drusco A, et al. 'Advanced' generation lentiviruses as efficient vectors for cardiomyocyte gene transduction in vitro and in vivo. Gene Ther. 2003;10:630–636
  14. Cockrell AS, Kafri T. Gene delivery by lentivirus vectors. Mol. Biotechnol. 2007;36:184–204
  15. Lundstrom K. Latest development in viral vectors for gene therapy. Trends Biotechnol. 2003;21:117–122
  16. Gregorevic P, Blankinship M, Allen J, Crawford R, Meuse L, Miller D, et al. Systemic delivery of genes to striated muscles using adeno-associated viral vectors. Nat. Med. 2004;10:828–834
  17. Kass-Eisler A, Falck-Pedersen E, Alvira M, Rivera J, Buttrick PM, Wittenberg BA, et al. Quantitative determination of adenovirus-mediated gene delivery to rat cardiac myocytes in vitro and in vivo. Proc. Natl. Acad. Sci. U. S. A. 1993;90:11498–11502
  18. Benjamin EJ, Wolf PA, D'Agostino RB, Silbershatz H, Kannel WB, Levy D. Impact of atrial fibrillation on the risk of death: the Framingham Heart Study. Circulation. 1998;98:946–952
  19. Amit G, Kikuchi K, Greener ID, Yang L, Novack V, Donahue JK. Selective Molecular Potassium Channel Blockade Prevents Atrial Fibrillation. Circulation. 2010;
  20. Curran ME, Splawski I, Timothy KW, Vincent GM, Green ED, Keating MT. A molecular basis for cardiac arrhythmia: HERG mutations cause long QT syndrome. Cell. 1995;80:795–803
  21. Burton DY, Song C, Fishbein I, Hazelwood S, Li Q, DeFelice S, et al. The incorporation of an ion channel gene mutation associated with the long QT syndrome (Q9E-hMiRP1) in a plasmid vector for site-specific arrhythmia gene therapy: in vitro and in vivo feasibility studies. Hum. Gene Ther. 2003;14:907–922
  22. Abbott GW, Sesti F, Splawski I, Buck ME, Lehmann MH, Timothy KW, et al. MiRP1 forms IKr potassium channels with HERG and is associated with cardiac arrhythmia. Cell. 1999;97:175–187
  23. Donahue JK, Heldman AW, Fraser H, McDonald AD, Miller JM, Rade JJ, et al. Focal modification of electrical conduction in the heart by viral gene transfer. Nat. Med. 2000;6:1395–1398
  24. Bauer A, McDonald AD, Nasir K, Peller L, Rade JJ, Miller JM, et al. Inhibitory G protein overexpression provides physiologically relevant heart rate control in persistent atrial fibrillation. Circulation. 2004;110:3115–3120
  25. Bunch TJ, Mahapatra S, Bruce GK, Johnson SB, Miller DV, Horne BD, et al. Impact of transforming growth factor-beta1 on atrioventricular node conduction modification by injected autologous fibroblasts in the canine heart. Circulation. 2006;113:2485–2494
  26. Rosenbaum DS, Jackson LE, Smith JM, Garan H, Ruskin JN, Cohen RJ. Electrical alternans and vulnerability to ventricular arrhythmias. N. Engl. J. Med. 1994;330:235–241
  27. Cutler MJ, Wan X, Laurita KR, Hajjar RJ, Rosenbaum DS. Targeted SERCA2a gene expression identifies molecular mechanism and therapeutic target for arrhythmogenic cardiac alternans. Circ. Arrhythm. Electrophysiol. 2009;2:686–694
  28. Peters NS, Coromilas J, Severs NJ, Wit AL. Disturbed connexin43 gap junction distribution correlates with the location of reentrant circuits in the epicardial border zone of healing canine infarcts that cause ventricular tachycardia. Circulation. 1997;95:988–996
  29. Lau DH, Clausen C, Sosunov EA, Shlapakova IN, Anyukhovsky EP, Danilo P, et al. Epicardial border zone overexpression of skeletal muscle sodium channel SkM1 normalizes activation, preserves conduction, and suppresses ventricular arrhythmia: an in silico, in vivo, in vitro study. Circulation. 2009;119:19–27
  30. Yang B, Lin H, Xiao J, Lu Y, Luo X, Li B, et al. The muscle-specific microRNA miR-1 regulates cardiac arrhythmogenic potential by targeting GJA1 and KCNJ2. Nat. Med. 2007;13:486–491
  31. Fishbein MC, Maclean D, Maroko PR. Experimental myocardial infarction in the rat: qualitative and quantitative changes during pathologic evolution. Am. J. Pathol. 1978;90:57–70
  32. Sasano T, McDonald AD, Kikuchi K, Donahue JK. Molecular ablation of ventricular tachycardia after myocardial infarction. Nat. Med. 2006;12:1256–1258
  33. Brunner M, Kodirov SA, Mitchell GF, Buckett PD, Shibata K, Folco EJ, et al. In vivo gene transfer of Kv1.5 normalizes action potential duration and shortens QT interval in mice with long QT phenotype. Am. J. Physiol. Heart Circ. Physiol. 2003;285:H194–H203
  34. Kodirov SA, Brunner M, Busconi L, Koren G. Long-term restitution of 4-aminopyridine-sensitive currents in Kv1DN ventricular myocytes using adeno-associated virus-mediated delivery of Kv1.5. FEBS Lett. 2003;550:74–78
  35. Edelberg JM, Aird WC, Rosenberg RD. Enhancement of murine cardiac chronotropy by the molecular transfer of the human beta2 adrenergic receptor cDNA. J. Clin. Invest. 1998;101:337–343
  36. Miake J, Marban E, Nuss HB. Biological pacemaker created by gene transfer. Nature. 2002;419:132–133
  37. Qu J, Plotnikov AN, Danilo P, Shlapakova I, Cohen IS, Robinson RB, et al. Expression and function of a biological pacemaker in canine heart. Circulation. 2003;107:1106–1109
  38. Plotnikov AN, Sosunov EA, Qu J, Shlapakova IN, Anyukhovsky EP, Liu L, et al. Biological pacemaker implanted in canine left bundle branch provides ventricular escape rhythms that have physiologically acceptable rates. Circulation. 2004;109:506–512
  39. Tse HF, Xue T, Lau CP, Siu CW, Wang K, Zhang QY, et al. 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. 2006;114:1000–1011
  40. Plotnikov AN, Shlapakova I, Szabolcs MJ, Danilo P, Lorell BH, Potapova IA, et al. Xenografted adult human mesenchymal stem cells provide a platform for sustained biological pacemaker function in canine heart. Circulation. 2007;116:706–713
  41. Kahlig KM, Saridey SK, Kaja A, Daniels MA, George AL, Wilson MH. Multiplexed transposon-mediated stable gene transfer in human cells. Proc. Natl. Acad. Sci. U. S. A. 2010;107:1343–1348
  42. Ying Y, Muller OJ, Goehringer C, Leuchs B, Trepel M, Katus HA, et al. Heart-targeted adeno-associated viral vectors selected by in vivo biopanning of a random viral display peptide library. Gene Ther. 2010;
  43. Cartier N, Hacein-Bey-Abina S, Bartholomae CC, Veres G, Schmidt M, Kutschera I, et al. Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science. 2009;326:818–823
  44. Aiuti A, Cattaneo F, Galimberti S, Benninghoff U, Cassani B, Callegaro L, et al. Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N. Engl. J. Med. 2009;360:447–458
  45. Bainbridge JW, Smith AJ, Barker SS, Robbie S, Henderson R, Balaggan K, et al. Effect of gene therapy on visual function in Leber's congenital amaurosis. N. Engl. J. Med. 2008;358:2231–2239

PII: S0022-2828(10)00289-0

doi: 10.1016/j.yjmcc.2010.07.022

Journal of Molecular and Cellular Cardiology
Volume 50, Issue 5 , Pages 759-765 , May 2011