Translational Research
Volume 156, Issue 3 , Pages 130-135 , September 2010

New therapies for the failing heart: trans-genes versus trans-cells

  • Vincenzo Lionetti

      Affiliations

    • Sector of Medicine, Scuola Superiore Sant'Anna, Pisa, Italy
    • Fondazione G. Monasterio Regione Toscana-CNR, Pisa, Italy
  • ,
  • Fabio A. Recchia

      Affiliations

    • Sector of Medicine, Scuola Superiore Sant'Anna, Pisa, Italy
    • Fondazione G. Monasterio Regione Toscana-CNR, Pisa, Italy
    • Department of Physiology, New York Medical College, Valhalla, NY
    • Corresponding Author InformationReprint requests: Fabio A Recchia, MD, PhD, Department of Physiology, New York Medical College, Valhalla, NY 10595

Received 12 May 2010 ,Revised 28 June 2010 ,Accepted 28 June 2010.

References 

  1. Packer M. The impossible task of developing a new treatment for heart failure. J Card Fail. 2002;8:193–196
  2. Capecchi MR. High efficiency transformation by direct microinjection of DNA into cultured mammalian cells. Cell. 1980;22:479–488
  3. Miller DA, Jolly DJ, Friedmann T, Verma IM. A transmissible retrovirus expressing human hypoxanthine phosphoribosyltransferase (HPRT): gene transfer into cells obtained from humans deficient in HPRT. Proc Natl Acad Sci U S A. 1983;80:4709–4713
  4. Miller AD, Eckner RJ, Jolly DJ, Friedmann T, Verma IM. Expression of a retrovirus encoding human HPRT in mice. Science. 1984;225:630–632
  5. Lin H, Parmacek MS, Morle G, Bolling S, Leiden JM. Expression of recombinant genes in myocardium in vivo after direct injection of DNA. Circulation. 1990;82:2217–2221
  6. Stewart DJ, Hilton JD, Arnold JM, 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;13:1503–1511
  7. Yang ZJ, Xu SL, Chen B, et al. Hepatocyte growth factor plays a critical role in the regulation of cytokine production and induction of endothelial progenitor cell mobilization: a pilot gene therapy study in patients with coronary heart disease. Clin Exp Pharmacol Physiol. 2009;36:790–796
  8. Stewart DJ, Kutryk MJ, Fitchett D, et al. VEGF gene therapy fails to improve perfusion of ischemic myocardium in patients with advanced coronary disease: results of the NORTHERN trial. Mol Ther. 2009;17:1109–1115
  9. Jackson KA, Majka SM, Wang H, et al. Regeneration of ischemic cardiac muscle and vascular endothelium by adult stem cells. J Clin Invest. 2001;107:1395–1402
  10. Kocher AA, Schuster MD, Szaboles MJ, et al. Neovascularization of ischemic myocardium by human bone-marrow derived angioblasts prevents cardiomyocyte apoptosis, reduces remodelling and improves cardiac function. Nat Med. 2001;7:430–436
  11. Menasche P, Hagege AA, Scorsin M, et al. Myoblast transplantation for heart failure. Lancet. 2001;357:279–280
  12. Orlic D, Kajstura J, Chimenti S, et al. Bone marrow cells regenerate infarcted myocardium. Nature. 2001;410:701–705
  13. Yoon PD, Kao RL, Magovern GJ. Myocardial regeneration. Transplanting satellite cells into damaged myocardium. Tex Heart Inst J. 1995;22:119–125
  14. Bittner RE, Schöfer C, Weipoltshammer K, et al. Recruitment of bone-marrow-derived cells by skeletal and cardiac muscle in adult dystrophic mdx mice. Anat Embryol. 1999;199:391–396
  15. Tomita S, Li RK, Weisel RD, et al. Autologous transplantation of bone marrow cells improves damaged heart function. Circulation. 1999;100:II247–II256
  16. Liechty KW, MacKenzie TC, Shaaban AF, et al. Human mesenchymal stem cells engraft and demonstrate site-specific differentiation after in utero transplantation in sheep. Nat Med. 2000;6:1282–1286
  17. Reinecke H, Minami E, Zhu WZ, Laflamme MA. Cardiogenic differentiation and transdifferentiation of progenitor cells. Circ Res. 2008;103:1058–1071
  18. Piepoli MF. Transplantation of progenitor cells and regeneration of damaged myocardium: more facts or doubts? Insights from experimental and clinical studies. J Cardiovasc Med. 2009;10:624–634
  19. Martin-Rendon E, Brunskill S, Doree C, et al. Stem cell treatment for acute myocardial infarction. Cochrane Database Syst Rev. 2008;CD006536
  20. Zhang SN, Sun AJ, Ge JB, et al. Intracoronary autologous bone marrow stem cells transfer for patients with acute myocardial infarction: A meta-analysis of randomised controlled trials. Int J Cardiol. 2009;136:178–185
  21. Schächinger V, Assmus B, Erbs S, et al. Intracoronary infusion of bone marrow-derived mononuclear cells abrogates adverse left ventricular remodelling post-acute myocardial infarction: insights from the reinfusion of enriched progenitor cells and infarct remodelling in acute myocardial infarction (REPAIR-AMI) trial. Eur J Heart Fail. 2009;11:973–979
  22. Murry CE, Soonpaa MH, Reinecke H, Schleyer H. Haematopoietic stem cells do not transdifferentiate into cardiac myocytes in myocardial infarcts. Nature. 2004;428:654–668
  23. Menasché P, Alfieri O, Janssens S, et al. The Myoblast Autologous Grafting in Ischemic Cardiomyopathy (MAGIC) trial: first randomized placebo-controlled study of myoblast transplantation. Circulation. 2008;117:1189–1200
  24. Smith RR, Barile L, Cho HC, et al. Regenerative potential of cardiosphere-derived cells expanded from percutaneous endomyocardial biopsy specimens. Circulation. 2007;115:896–908
  25. Tang XL, Rokosh G, Sanganalmath SK, et al. Intracoronary administration of cardiac progenitor cells alleviates left ventricular dysfunction in rats with a 30-day-old infarction. Circulation. 2010;121:293–305
  26. Hare JM, Traverse JH, Henry TD, et al. A randomized, double-blind, placebo-controlled, dose-escalation study of intravenous adult human mesenchymal stem cells (prochymal) after acute myocardial infarction. J Am Coll Cardiol. 2009;54:2277–2286
  27. Madonna R, De Caterina R. Adipose tissue: a new source for cardiovascular repair. J Cardiovasc Med. 2010;11:71–80
  28. Ventura C, Cantoni S, Bianchi F, et al. Hyaluronan mixed esters of butyric and retinoic Acid drive cardiac and endothelial fate in term placenta human mesenchymal stem cells and enhance cardiac repair in infarcted rat hearts. J Biol Chem. 2007;282:14243–14252
  29. Vinge LE, Raake PW, Koch WJ. Gene therapy in heart failure. Circ Res. 2008;102:1458–1470
  30. Byrne MJ, Power JM, Preovolos A, Mariani JA, Hajjar RJ, Kaye DM. Recirculating cardiac delivery of AAV2/1SERCA2a improves myocardial function in an experimental model of heart failure in large animals. Gene Ther. 2008;15:1550–1557
  31. Wittköpper K, Fabritz L, Neef S, et al. Constitutively active phosphatase inhibitor-1 improves cardiac contractility in young mice but is deleterious after catecholaminergic stress and with aging. J Clin Invest. 2010;120:617–626
  32. Liu Q, Chen X, Macdonnell SM, et al. Protein kinase C{alpha}, but not PKC{beta} or PKC{gamma}, regulates contractility and heart failure susceptibility: implications for ruboxistaurin as a novel therapeutic approach. Circ Res. 2009;105:194–200
  33. Jaski BE, Jessup ML, Mancini DM, et al. 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. 2009;15:171–181
  34. Mangi AA, Noiseux N, Kong D, et al. Mesenchymal stem cells modified with Akt prevent remodeling and restore performance of infarcted hearts. Nat Med. 2003;9:1195–1201
  35. Matsumoto R, Omura T, Yoshiyama M, et al. Vascular endothelial growth factor-expressing mesenchymal stem cell transplantation for the treatment of acute myocardial infarction. Arterioscler Thromb Vasc Biol. 2005;25:1168–1173
  36. van Til NP, Stok M, Aerts Kaya FS, et al. Lentiviral gene therapy of murine hematopoietic stem cells ameliorates the Pompe disease phenotype. Blood. 2010;115:5329–5337
  37. Gnecchi M, Zhang Z, Ni A, Dzau VJ. Paracrine mechanisms in adult stem cell signaling and therapy. Circ Res. 2008;103:1204–1219
  38. Burchfield JS, Dimmeler S. Role of paracrine factors in stem and progenitor cell mediated cardiac repair and tissue fibrosis. Fibrogenesis Tissue Repair. 2008;1:4
  39. 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;98:954–961
  40. Zentilin L, Puligadda U, Lionetti V, et al. Cardiomyocyte VEGFR-1 activation by VEGF-B induces compensatory hypertrophy and preserves cardiac function after myocardial infarction. FASEB J. 2010;24:1467–1478
  41. Pepe M, Mamdani M, Zentilin L, et al. Intramyocardial VEGF-B167 gene delivery delays the progression towards congestive failure in dogs with pacing-induced dilated cardiomyopathy. Circ Res. 2010;106:1893–1903
  42. Everly MJ. Cardiac transplantation in the United States: an analysis of the UNOS registry. Clin Transpl. 2008;35–43
  43. Gupta S, Maitra R, Young D, Gupta A, Sen S. Silencing the myotrophin gene by RNA interference leads to the regression of cardiac hypertrophy. Am J Physiol Heart Circ Physiol. 2009;297:H627–H636
  44. Suckau L, Fechner H, Chemaly E, et al. Long-term cardiac-targeted RNA interference for the treatment of heart failure restores cardiac function and reduces pathological hypertrophy. Circulation. 2009;119:1241–1252
  45. Ye Y, Hu Z, Lin Y, Zhang C, Perez-Polo JR. Downregulation of microRNA-29 by antisense inhibitors and a PPAR-{gamma} agonist protects against myocardial ischaemia-reperfusion injury. Cardiovasc Res. 2010 Mar 13;[Epub ahead of print]
  46. Leor J, Tuvia S, Guetta V, et al. Intracoronary injection of in situ forming alginate hydrogel reverses left ventricular remodeling after myocardial infarction in Swine. J Am Coll Cardiol. 2009;54:1014–1023
  47. Lionetti V, Cantoni S, Cavallini C, et al. Hyaluronan mixed esters of butyric and retinoic acid affording myocardial survival and repair without stem cell transplantation. J Biol Chem. 2010;285:9949–9961

PII: S1931-5244(10)00124-6

doi: 10.1016/j.trsl.2010.06.009

Translational Research
Volume 156, Issue 3 , Pages 130-135 , September 2010