Skip to main content
Top

2013 | OriginalPaper | Hoofdstuk

44. Cardiovascular Gene Therapy

Auteurs : Thomas J. LaRocca, MD, PhD, Roger J. Hajjar, MD

Gepubliceerd in: Essential Cardiology

Uitgeverij: Springer New York

share
DELEN

Deel dit onderdeel of sectie (kopieer de link)

  • Optie A:
    Klik op de rechtermuisknop op de link en selecteer de optie “linkadres kopiëren”
  • Optie B:
    Deel de link per e-mail

Abstract

Gene therapy is rapidly evolving as an effective therapeutic strategy for patients suffering from various forms of cardiovascular disease. Since the conceptualization of gene therapy, the scientific and clinical community has learned a great deal from the successes and, unfortunately, failures of previous gene therapy clinical trials over the past decades. Cardiovascular gene therapy has benefitted from recent advancements in vector technology, design and vector delivery modalities. Nonviral and viral vectors including plasmids, retrovirus, lentivirus, adenovirus, and adeno-associated virus each have unique cellular transduction mechanisms and expression profiles. Due to the exclusive qualities of each vector, the selection and development of vectors for cardiovascular gene therapy is critically dependent on the specific cardiac pathology. In addition, how these vectors are delivered to the heart greatly affects myocardial transduction and clinical outcomes. It is necessary to employ the proper vector and delivery modality that can safely and efficiently transduce the myocardium with well-validated transgenes shown to protect against acute ischemia, arrhythmias, hypertrophy, or ventricular failure. Gene therapies targeting the β-adrenergic pathway, calcium regulatory mechanisms, angiogenesis, or the CXCL12/CXCR4 axis have shown promise both preclinically and in human clinical trials. The current state of the art of cardiovascular gene therapy as well as clinically significant molecular targets is reviewed as this promising strategy enters the clinical realm.
Literatuur
1.
go back to reference Yockman JW et al. Novel polymer carriers and gene constructs for treatment of myocardial ischemia and infarction. J Control Release. 2008;132(3):260–6.PubMedCrossRef Yockman JW et al. Novel polymer carriers and gene constructs for treatment of myocardial ischemia and infarction. J Control Release. 2008;132(3):260–6.PubMedCrossRef
2.
go back to reference Wasala NB, Shin JH, Duan D. The evolution of heart gene delivery vectors. J Gene Med. 2011;13(10):557–65.PubMedCrossRef Wasala NB, Shin JH, Duan D. The evolution of heart gene delivery vectors. J Gene Med. 2011;13(10):557–65.PubMedCrossRef
3.
go back to reference Harraghy N, Gaussin A, Mermod N. Sustained transgene expression using MAR elements. Curr Gene Ther. 2008;8(5):353–66.PubMedCrossRef Harraghy N, Gaussin A, Mermod N. Sustained transgene expression using MAR elements. Curr Gene Ther. 2008;8(5):353–66.PubMedCrossRef
4.
go back to reference Girod PA et al. Genome-wide prediction of matrix attachment regions that increase gene expression in mammalian cells. Nat Methods. 2007;4(9):747–53.PubMedCrossRef Girod PA et al. Genome-wide prediction of matrix attachment regions that increase gene expression in mammalian cells. Nat Methods. 2007;4(9):747–53.PubMedCrossRef
5.
go back to reference Ehrhardt A et al. Optimization of cis-acting elements for gene expression from nonviral vectors in vivo. Hum Gene Ther. 2003;14(3):215–25.PubMedCrossRef Ehrhardt A et al. Optimization of cis-acting elements for gene expression from nonviral vectors in vivo. Hum Gene Ther. 2003;14(3):215–25.PubMedCrossRef
6.
go back to reference Argyros O et al. Development of S/MAR minicircles for enhanced and persistent transgene expression in the mouse liver. J Mol Med (Berl). 2011;89(5):515–29.CrossRef Argyros O et al. Development of S/MAR minicircles for enhanced and persistent transgene expression in the mouse liver. J Mol Med (Berl). 2011;89(5):515–29.CrossRef
7.
go back to reference Themis M et al. Mutational effects of retrovirus insertion on the genome of V79 cells by an attenuated retrovirus vector: implications for gene therapy. Gene Ther. 2003;10(19):1703–11.PubMedCrossRef Themis M et al. Mutational effects of retrovirus insertion on the genome of V79 cells by an attenuated retrovirus vector: implications for gene therapy. Gene Ther. 2003;10(19):1703–11.PubMedCrossRef
8.
go back to reference Fischer A, Hacein-Bey-Abina S, Cavazzana-Calvo M. 20 years of gene therapy for SCID. Nat Immunol. 2010;11(6):457–60.PubMedCrossRef Fischer A, Hacein-Bey-Abina S, Cavazzana-Calvo M. 20 years of gene therapy for SCID. Nat Immunol. 2010;11(6):457–60.PubMedCrossRef
9.
go back to reference Hacein-Bey-Abina S et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science. 2003;302(5644):415–9.PubMedCrossRef Hacein-Bey-Abina S et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science. 2003;302(5644):415–9.PubMedCrossRef
10.
go back to reference Cavazzana-Calvo M et al. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science. 2000;288(5466):669–72.PubMedCrossRef Cavazzana-Calvo M et al. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science. 2000;288(5466):669–72.PubMedCrossRef
11.
go back to reference Kraunus J et al. Self-inactivating retroviral vectors with improved RNA processing. Gene Ther. 2004;11(21):1568–78.PubMedCrossRef Kraunus J et al. Self-inactivating retroviral vectors with improved RNA processing. Gene Ther. 2004;11(21):1568–78.PubMedCrossRef
12.
go back to reference Vodicka MA. Determinants for lentiviral infection of non-dividing cells. Somat Cell Mol Genet. 2001;26(1–6):35–49.PubMedCrossRef Vodicka MA. Determinants for lentiviral infection of non-dividing cells. Somat Cell Mol Genet. 2001;26(1–6):35–49.PubMedCrossRef
13.
go back to reference Levine BL et al. Gene transfer in humans using a conditionally replicating lentiviral vector. Proc Natl Acad Sci USA. 2006;103(46): 17372–7.PubMedCrossRef Levine BL et al. Gene transfer in humans using a conditionally replicating lentiviral vector. Proc Natl Acad Sci USA. 2006;103(46): 17372–7.PubMedCrossRef
15.
go back to reference Mortellaro A et al. Ex vivo gene therapy with lentiviral vectors rescues adenosine deaminase (ADA)-deficient mice and corrects their immune and metabolic defects. Blood. 2006;108(9):2979–88.PubMedCrossRef Mortellaro A et al. Ex vivo gene therapy with lentiviral vectors rescues adenosine deaminase (ADA)-deficient mice and corrects their immune and metabolic defects. Blood. 2006;108(9):2979–88.PubMedCrossRef
16.
go back to reference Lyle C, McCormick F. Integrin alphavbeta5 is a primary receptor for adenovirus in CAR-negative cells. Virol J. 2010;7:148.PubMedCrossRef Lyle C, McCormick F. Integrin alphavbeta5 is a primary receptor for adenovirus in CAR-negative cells. Virol J. 2010;7:148.PubMedCrossRef
17.
go back to reference Matyas L et al. Arteriogenic gene therapy in patients with unreconstructable critical limb ischemia: a randomized, placebo-controlled clinical trial of adenovirus 5-delivered fibroblast growth factor-4. Hum Gene Ther. 2005;16(10):1202–11.PubMedCrossRef Matyas L et al. Arteriogenic gene therapy in patients with unreconstructable critical limb ischemia: a randomized, placebo-controlled clinical trial of adenovirus 5-delivered fibroblast growth factor-4. Hum Gene Ther. 2005;16(10):1202–11.PubMedCrossRef
18.
go back to reference Tongers J, Roncalli JG, Losordo DW. Therapeutic angiogenesis for critical limb ischemia: microvascular therapies coming of age. Circulation. 2008;118(1):9–16.PubMedCrossRef Tongers J, Roncalli JG, Losordo DW. Therapeutic angiogenesis for critical limb ischemia: microvascular therapies coming of age. Circulation. 2008;118(1):9–16.PubMedCrossRef
19.
go back to reference Raper SE et al. Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer. Mol Genet Metab. 2003;80(1–2):148–58.PubMedCrossRef Raper SE et al. Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer. Mol Genet Metab. 2003;80(1–2):148–58.PubMedCrossRef
20.
go back to reference Becerra SP et al. Synthesis of adeno-associated virus structural proteins requires both alternative mRNA splicing and alternative initiations from a single transcript. J Virol. 1988;62(8):2745–54.PubMed Becerra SP et al. Synthesis of adeno-associated virus structural proteins requires both alternative mRNA splicing and alternative initiations from a single transcript. J Virol. 1988;62(8):2745–54.PubMed
21.
go back to reference Pacak CA, Byrne BJ. AAV vectors for cardiac gene transfer: experimental tools and clinical opportunities. Mol Ther. 2011;19(9):1582–90.PubMedCrossRef Pacak CA, Byrne BJ. AAV vectors for cardiac gene transfer: experimental tools and clinical opportunities. Mol Ther. 2011;19(9):1582–90.PubMedCrossRef
22.
go back to reference Asokan A et al. Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle. Nat Biotechnol. 2010;28(1):79–82.PubMedCrossRef Asokan A et al. Reengineering a receptor footprint of adeno-­associated virus enables selective and systemic gene transfer to muscle. Nat Biotechnol. 2010;28(1):79–82.PubMedCrossRef
23.
go back to reference Wang J, Faust SM, Rabinowitz JE. The next step in gene delivery: molecular engineering of adeno-associated virus serotypes. J Mol Cell Cardiol. 2011;50(5):793–802.PubMedCrossRef Wang J, Faust SM, Rabinowitz JE. The next step in gene delivery: molecular engineering of adeno-associated virus serotypes. J Mol Cell Cardiol. 2011;50(5):793–802.PubMedCrossRef
24.
go back to reference Mitchell AM et al. AAV’s anatomy: roadmap for optimizing vectors for translational success. Curr Gene Ther. 2010;10(5):319–40.PubMedCrossRef Mitchell AM et al. AAV’s anatomy: roadmap for optimizing vectors for translational success. Curr Gene Ther. 2010;10(5):319–40.PubMedCrossRef
25.
go back to reference Li W et al. Engineering and selection of shuffled AAV genomes: a new strategy for producing targeted biological nanoparticles. Mol Ther. 2008;16(7):1252–60.PubMedCrossRef Li W et al. Engineering and selection of shuffled AAV genomes: a new strategy for producing targeted biological nanoparticles. Mol Ther. 2008;16(7):1252–60.PubMedCrossRef
26.
go back to reference Ying Y et al. Heart-targeted adeno-associated viral vectors selected by in vivo biopanning of a random viral display peptide library. Gene Ther. 2010;17(8):980–90.PubMedCrossRef Ying Y et al. Heart-targeted adeno-associated viral vectors selected by in vivo biopanning of a random viral display peptide library. Gene Ther. 2010;17(8):980–90.PubMedCrossRef
27.
go back to reference Jaski BE 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(3):171–81.PubMedCrossRef Jaski BE 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(3):171–81.PubMedCrossRef
28.
go back to reference Nonnenmacher M, Weber T. Adeno-associated virus 2 infection requires endocytosis through the CLIC/GEEC pathway. Cell Host Microbe. 2011;10(6):563–76.PubMedCrossRef Nonnenmacher M, Weber T. Adeno-associated virus 2 infection requires endocytosis through the CLIC/GEEC pathway. Cell Host Microbe. 2011;10(6):563–76.PubMedCrossRef
29.
go back to reference Nonnenmacher M, Weber T. Intracellular transport of recombinant adeno-associated virus vectors. Gene Ther. 2012;19(6):649–58.PubMedCrossRef Nonnenmacher M, Weber T. Intracellular transport of recombinant adeno-associated virus vectors. Gene Ther. 2012;19(6):649–58.PubMedCrossRef
30.
go back to reference Boecker W et al. Cardiac-specific gene expression facilitated by an enhanced myosin light chain promoter. Mol Imaging. 2004;3(2):69–75.PubMedCrossRef Boecker W et al. Cardiac-specific gene expression facilitated by an enhanced myosin light chain promoter. Mol Imaging. 2004;3(2):69–75.PubMedCrossRef
31.
go back to reference Prasad KM et al. Robust cardiomyocyte-specific gene expression following systemic injection of AAV: in vivo gene delivery follows a Poisson distribution. Gene Ther. 2011;18(1):43–52.PubMedCrossRef Prasad KM et al. Robust cardiomyocyte-specific gene expression following systemic injection of AAV: in vivo gene delivery follows a Poisson distribution. Gene Ther. 2011;18(1):43–52.PubMedCrossRef
32.
go back to reference Ruan H et al. A hypoxia-regulated adeno-associated virus vector for cancer-specific gene therapy. Neoplasia. 2001;3(3):255–63.PubMedCrossRef Ruan H et al. A hypoxia-regulated adeno-associated virus vector for cancer-specific gene therapy. Neoplasia. 2001;3(3):255–63.PubMedCrossRef
33.
go back to reference Su H, Kan YW. Adeno-associated viral vector-delivered hypoxia-inducible gene expression in ischemic hearts. Methods Mol Biol. 2007;366:331–42.PubMedCrossRef Su H, Kan YW. Adeno-associated viral vector-delivered hypoxia-inducible gene expression in ischemic hearts. Methods Mol Biol. 2007;366:331–42.PubMedCrossRef
34.
go back to reference Vanrell L et al. Development of a liver-specific Tet-on inducible system for AAV vectors and its application in the treatment of liver cancer. Mol Ther. 2011;19(7):1245–53.PubMedCrossRef Vanrell L et al. Development of a liver-specific Tet-on inducible system for AAV vectors and its application in the treatment of liver cancer. Mol Ther. 2011;19(7):1245–53.PubMedCrossRef
35.
go back to reference Ye X et al. Regulated delivery of therapeutic proteins after in vivo somatic cell gene transfer. Science. 1999;283(5398):88–91.PubMedCrossRef Ye X et al. Regulated delivery of therapeutic proteins after in vivo somatic cell gene transfer. Science. 1999;283(5398):88–91.PubMedCrossRef
36.
go back to reference Chen ZY et al. Ultrasound- and liposome microbubble-mediated targeted gene transfer to cardiomyocytes in vivo accompanied by polyethylenimine. J Ultrasound Med. 2011;30(9):1247–58.PubMed Chen ZY et al. Ultrasound- and liposome microbubble-mediated targeted gene transfer to cardiomyocytes in vivo accompanied by polyethylenimine. J Ultrasound Med. 2011;30(9):1247–58.PubMed
37.
go back to reference Mariani JA, Kaye DM. Delivery of gene and cellular therapies for heart disease. J Cardiovasc Transl Res. 2010;3(4):417–26.PubMedCrossRef Mariani JA, Kaye DM. Delivery of gene and cellular therapies for heart disease. J Cardiovasc Transl Res. 2010;3(4):417–26.PubMedCrossRef
38.
go back to reference Parsa CJ et al. Catheter-mediated subselective intracoronary gene delivery to the rabbit heart: introduction of a novel method. J Gene Med. 2005;7(5):595–603.PubMedCrossRef Parsa CJ et al. Catheter-mediated subselective intracoronary gene delivery to the rabbit heart: introduction of a novel method. J Gene Med. 2005;7(5):595–603.PubMedCrossRef
39.
go back to reference Sasano T et al. Targeted high-efficiency, homogeneous myocardial gene transfer. J Mol Cell Cardiol. 2007;42(5):954–61.PubMedCrossRef Sasano T et al. Targeted high-efficiency, homogeneous myocardial gene transfer. J Mol Cell Cardiol. 2007;42(5):954–61.PubMedCrossRef
40.
go back to reference Raake PW et al. Cardio-specific long-term gene expression in a porcine model after selective pressure-regulated retroinfusion of adeno-associated viral (AAV) vectors. Gene Ther. 2008;15(1):12–7.PubMedCrossRef Raake PW et al. Cardio-specific long-term gene expression in a porcine model after selective pressure-regulated retroinfusion of adeno-associated viral (AAV) vectors. Gene Ther. 2008;15(1):12–7.PubMedCrossRef
41.
go back to reference Karakikes I. Concomitant intravenous nitroglycerin with intracoronary delivery of AAV1.SERCA2a enhances gene transfer in porcine hearts. Mol Ther. 2012;20:565–71.PubMedCrossRef Karakikes I. Concomitant intravenous nitroglycerin with intracoronary delivery of AAV1.SERCA2a enhances gene transfer in porcine hearts. Mol Ther. 2012;20:565–71.PubMedCrossRef
42.
go back to reference Kaye DM et al. Percutaneous cardiac recirculation-mediated gene transfer of an inhibitory phospholamban peptide reverses advanced heart failure in large animals. J Am Coll Cardiol. 2007;50(3):253–60.PubMedCrossRef Kaye DM et al. Percutaneous cardiac recirculation-mediated gene transfer of an inhibitory phospholamban peptide reverses advanced heart failure in large animals. J Am Coll Cardiol. 2007;50(3):253–60.PubMedCrossRef
43.
go back to reference Byrne MJ et al. Recirculating cardiac delivery of AAV2/1SERCA2a improves myocardial function in an experimental model of heart failure in large animals. Gene Ther. 2008;15(23):1550–7.PubMedCrossRef Byrne MJ et al. Recirculating cardiac delivery of AAV2/1SERCA2a improves myocardial function in an experimental model of heart failure in large animals. Gene Ther. 2008;15(23):1550–7.PubMedCrossRef
44.
go back to reference Fuchs S et al. A randomized, double-blind, placebo-controlled, multicenter, pilot study of the safety and feasibility of catheter-based intramyocardial injection of AdVEGF121 in patients with refractory advanced coronary artery disease. Catheter Cardiovasc Interv. 2006;68(3):372–8.PubMedCrossRef Fuchs S et al. A randomized, double-blind, placebo-controlled, multicenter, pilot study of the safety and feasibility of catheter-based intramyocardial injection of AdVEGF121 in patients with refractory advanced coronary artery disease. Catheter Cardiovasc Interv. 2006;68(3):372–8.PubMedCrossRef
45.
go back to reference Losordo DW et al. Intramyocardial, autologous CD34+ cell therapy for refractory angina. Circ Res. 2011;109(4):428–36.PubMedCrossRef Losordo DW et al. Intramyocardial, autologous CD34+ cell therapy for refractory angina. Circ Res. 2011;109(4):428–36.PubMedCrossRef
46.
go back to reference Baldazzi F et al. Release of biomarkers of myocardial damage after direct intramyocardial injection of genes and stem cells via the percutaneous transluminal route. Eur Heart J. 2008;29(15):1819–26.PubMedCrossRef Baldazzi F et al. Release of biomarkers of myocardial damage after direct intramyocardial injection of genes and stem cells via the percutaneous transluminal route. Eur Heart J. 2008;29(15):1819–26.PubMedCrossRef
47.
go back to reference Ishikawa K et al. Sterile abscess in the myocardium after direct intramyocardial injection related to gene therapy in a Swine model. ISRN Cardiol. 2011;2011:319453.PubMed Ishikawa K et al. Sterile abscess in the myocardium after direct intramyocardial injection related to gene therapy in a Swine model. ISRN Cardiol. 2011;2011:319453.PubMed
48.
go back to reference Ladage D et al. Delivery of gelfoam-enabled cells and vectors into the pericardial space using a percutaneous approach in a porcine model. Gene Ther. 2011;18(10):979–85.PubMedCrossRef Ladage D et al. Delivery of gelfoam-enabled cells and vectors into the pericardial space using a percutaneous approach in a porcine model. Gene Ther. 2011;18(10):979–85.PubMedCrossRef
49.
go back to reference Aragon JP et al. Beta3-adrenoreceptor stimulation ameliorates myocardial ischemia-reperfusion injury via endothelial nitric oxide synthase and neuronal nitric oxide synthase activation. J Am Coll Cardiol. 2011;58(25):2683–91.PubMedCrossRef Aragon JP et al. Beta3-adrenoreceptor stimulation ameliorates myocardial ischemia-reperfusion injury via endothelial nitric oxide synthase and neuronal nitric oxide synthase activation. J Am Coll Cardiol. 2011;58(25):2683–91.PubMedCrossRef
50.
go back to reference Calvert JW et al. Exercise protects against myocardial ischemia-reperfusion injury via stimulation of beta(3)-adrenergic receptors and increased nitric oxide signaling: role of nitrite and nitrosothiols. Circ Res. 2011;108(12):1448–58.PubMedCrossRef Calvert JW et al. Exercise protects against myocardial ischemia-reperfusion injury via stimulation of beta(3)-adrenergic receptors and increased nitric oxide signaling: role of nitrite and nitrosothiols. Circ Res. 2011;108(12):1448–58.PubMedCrossRef
51.
go back to reference Morisco C et al. Beta-adrenergic cardiac hypertrophy is mediated primarily by the beta(1)-subtype in the rat heart. J Mol Cell Cardiol. 2001;33(3):561–73.PubMedCrossRef Morisco C et al. Beta-adrenergic cardiac hypertrophy is mediated primarily by the beta(1)-subtype in the rat heart. J Mol Cell Cardiol. 2001;33(3):561–73.PubMedCrossRef
52.
go back to reference Woo AY, Xiao RP. beta-Adrenergic receptor subtype signaling in heart: from bench to bedside. Acta Pharmacol Sin. 2012;33(3):335–41.PubMedCrossRef Woo AY, Xiao RP. beta-Adrenergic receptor subtype signaling in heart: from bench to bedside. Acta Pharmacol Sin. 2012;33(3):335–41.PubMedCrossRef
53.
go back to reference Zhu W. beta-adrenergic receptor subtype signaling in the heart: from bench to the bedside. Curr Top Membr. 2011;67:191–204.PubMedCrossRef Zhu W. beta-adrenergic receptor subtype signaling in the heart: from bench to the bedside. Curr Top Membr. 2011;67:191–204.PubMedCrossRef
54.
go back to reference Patel PA, Tilley DG, Rockman HA. Physiologic and cardiac roles of beta-arrestins. J Mol Cell Cardiol. 2009;46(3):300–8.PubMedCrossRef Patel PA, Tilley DG, Rockman HA. Physiologic and cardiac roles of beta-arrestins. J Mol Cell Cardiol. 2009;46(3):300–8.PubMedCrossRef
55.
go back to reference Tilley DG. beta-Arrestin mediates beta1-adrenergic receptor-epidermal growth factor receptor interaction and downstream signaling. J Biol Chem. 2009;284(30):20375–86.PubMedCrossRef Tilley DG. beta-Arrestin mediates beta1-adrenergic receptor-­epidermal growth factor receptor interaction and downstream ­signaling. J Biol Chem. 2009;284(30):20375–86.PubMedCrossRef
56.
go back to reference Noma T et al. Beta-arrestin-mediated beta1-adrenergic receptor transactivation of the EGFR confers cardioprotection. J Clin Invest. 2007;117(9):2445–58.PubMedCrossRef Noma T et al. Beta-arrestin-mediated beta1-adrenergic receptor transactivation of the EGFR confers cardioprotection. J Clin Invest. 2007;117(9):2445–58.PubMedCrossRef
57.
go back to reference Huang ZM, Gold JI, Koch WJ. G protein-coupled receptor kinases in normal and failing myocardium. Front Biosci. 2012;17:3047–60.CrossRef Huang ZM, Gold JI, Koch WJ. G protein-coupled receptor kinases in normal and failing myocardium. Front Biosci. 2012;17:3047–60.CrossRef
58.
go back to reference Akhter SA et al. In vivo inhibition of elevated myocardial beta-adrenergic receptor kinase activity in hybrid transgenic mice restores normal beta-adrenergic signaling and function. Circulation. 1999;100(6):648–53.PubMedCrossRef Akhter SA et al. In vivo inhibition of elevated myocardial beta-adrenergic receptor kinase activity in hybrid transgenic mice restores normal beta-adrenergic signaling and function. Circulation. 1999;100(6):648–53.PubMedCrossRef
59.
go back to reference Raake PW et al. AAV6.betaARKct cardiac gene therapy ameliorates cardiac function and normalizes the catecholaminergic axis in a clinically relevant large animal heart failure model. Eur Heart J. 2013;34(19):1437–47. Raake PW et al. AAV6.betaARKct cardiac gene therapy ameliorates cardiac function and normalizes the catecholaminergic axis in a clinically relevant large animal heart failure model. Eur Heart J. 2013;34(19):1437–47.
60.
go back to reference Williams ML et al. Targeted beta-adrenergic receptor kinase (betaARK1) inhibition by gene transfer in failing human hearts. Circulation. 2004;109(13):1590–3.PubMedCrossRef Williams ML et al. Targeted beta-adrenergic receptor kinase (betaARK1) inhibition by gene transfer in failing human hearts. Circulation. 2004;109(13):1590–3.PubMedCrossRef
61.
62.
go back to reference Okumura S et al. Disruption of type 5 adenylyl cyclase gene preserves cardiac function against pressure overload. Proc Natl Acad Sci USA. 2003;100(17):9986–90.PubMedCrossRef Okumura S et al. Disruption of type 5 adenylyl cyclase gene preserves cardiac function against pressure overload. Proc Natl Acad Sci USA. 2003;100(17):9986–90.PubMedCrossRef
63.
go back to reference Rebolledo B et al. Adenylylcyclase gene transfer increases function of the failing heart. Hum Gene Ther. 2006;17(10):1043–8.PubMedCrossRef Rebolledo B et al. Adenylylcyclase gene transfer increases function of the failing heart. Hum Gene Ther. 2006;17(10):1043–8.PubMedCrossRef
64.
go back to reference Gao MH, Hammond HK. Unanticipated signaling events associated with cardiac adenylyl cyclase gene transfer. J Mol Cell Cardiol. 2011;50(5):751–8.PubMedCrossRef Gao MH, Hammond HK. Unanticipated signaling events associated with cardiac adenylyl cyclase gene transfer. J Mol Cell Cardiol. 2011;50(5):751–8.PubMedCrossRef
65.
go back to reference Lai NC et al. Intracoronary adenovirus encoding adenylyl cyclase VI increases left ventricular function in heart failure. Circulation. 2004;110(3):330–6.PubMedCrossRef Lai NC et al. Intracoronary adenovirus encoding adenylyl cyclase VI increases left ventricular function in heart failure. Circulation. 2004;110(3):330–6.PubMedCrossRef
66.
go back to reference Hajjar RJ et al. Physiological effects of adenoviral gene transfer of sarcoplasmic reticulum calcium ATPase in isolated rat myocytes. Circulation. 1997;95(2):423–9.PubMedCrossRef Hajjar RJ et al. Physiological effects of adenoviral gene transfer of sarcoplasmic reticulum calcium ATPase in isolated rat myocytes. Circulation. 1997;95(2):423–9.PubMedCrossRef
67.
go back to reference del Monte F et al. Restoration of contractile function in isolated cardiomyocytes from failing human hearts by gene transfer of SERCA2a. Circulation. 1999;100(23):2308–11.CrossRef del Monte F et al. Restoration of contractile function in isolated cardiomyocytes from failing human hearts by gene transfer of SERCA2a. Circulation. 1999;100(23):2308–11.CrossRef
68.
go back to reference Kawase Y et al. Reversal of cardiac dysfunction after long-term expression of SERCA2a by gene transfer in a pre-clinical model of heart failure. J Am Coll Cardiol. 2008;51(11):1112–9.PubMedCrossRef Kawase Y et al. Reversal of cardiac dysfunction after long-term expression of SERCA2a by gene transfer in a pre-clinical model of heart failure. J Am Coll Cardiol. 2008;51(11):1112–9.PubMedCrossRef
69.
go back to reference Prunier F et al. Prevention of ventricular arrhythmias with sarcoplasmic reticulum Ca2+ ATPase pump overexpression in a porcine model of ischemia reperfusion. Circulation. 2008;118(6):614–24.PubMedCrossRef Prunier F et al. Prevention of ventricular arrhythmias with sarcoplasmic reticulum Ca2+ ATPase pump overexpression in a porcine model of ischemia reperfusion. Circulation. 2008;118(6):614–24.PubMedCrossRef
70.
go back to reference Jessup M et al. Calcium Upregulation by Percutaneous Administration of Gene Therapy in Cardiac Disease (CUPID): a phase 2 trial of intracoronary gene therapy of sarcoplasmic reticulum Ca2+−ATPase in patients with advanced heart failure. Circulation. 2011;124(3):304–13.PubMedCrossRef Jessup M et al. Calcium Upregulation by Percutaneous Administration of Gene Therapy in Cardiac Disease (CUPID): a phase 2 trial of intracoronary gene therapy of sarcoplasmic reticulum Ca2+−ATPase in patients with advanced heart failure. Circulation. 2011;124(3):304–13.PubMedCrossRef
71.
go back to reference Kho C et al. SUMO1-dependent modulation of SERCA2a in heart failure. Nature. 2011;477(7366):601–5.PubMedCrossRef Kho C et al. SUMO1-dependent modulation of SERCA2a in heart failure. Nature. 2011;477(7366):601–5.PubMedCrossRef
72.
73.
go back to reference Wright NT et al. S100A1: structure, function, and therapeutic potential. Curr Chem Biol. 2009;3(2):138–45.PubMed Wright NT et al. S100A1: structure, function, and therapeutic potential. Curr Chem Biol. 2009;3(2):138–45.PubMed
74.
go back to reference Kiewitz R et al. Transcriptional regulation of S100A1 and expression during mouse heart development. Biochim Biophys Acta. 2000;1498(2–3):207–19.PubMedCrossRef Kiewitz R et al. Transcriptional regulation of S100A1 and expression during mouse heart development. Biochim Biophys Acta. 2000;1498(2–3):207–19.PubMedCrossRef
75.
go back to reference Remppis A et al. Altered expression of the Ca(2+)-binding protein S100A1 in human cardiomyopathy. Biochim Biophys Acta. 1996;1313(3):253–7.PubMedCrossRef Remppis A et al. Altered expression of the Ca(2+)-binding protein S100A1 in human cardiomyopathy. Biochim Biophys Acta. 1996;1313(3):253–7.PubMedCrossRef
76.
go back to reference Remppis A et al. The small EF-hand Ca2+ binding protein S100A1 increases contractility and Ca2+ cycling in rat cardiac myocytes. Basic Res Cardiol. 2002;97 Suppl 1:I56–62.PubMed Remppis A et al. The small EF-hand Ca2+ binding protein S100A1 increases contractility and Ca2+ cycling in rat cardiac myocytes. Basic Res Cardiol. 2002;97 Suppl 1:I56–62.PubMed
77.
go back to reference Yamasaki R et al. Titin-actin interaction in mouse myocardium: passive tension modulation and its regulation by calcium/S100A1. Biophys J. 2001;81(4):2297–313.PubMedCrossRef Yamasaki R et al. Titin-actin interaction in mouse myocardium: passive tension modulation and its regulation by calcium/S100A1. Biophys J. 2001;81(4):2297–313.PubMedCrossRef
78.
go back to reference Brinks H et al. S100A1 genetically targeted therapy reverses dysfunction of human failing cardiomyocytes. J Am Coll Cardiol. 2011;58(9):966–73.PubMedCrossRef Brinks H et al. S100A1 genetically targeted therapy reverses dysfunction of human failing cardiomyocytes. J Am Coll Cardiol. 2011;58(9):966–73.PubMedCrossRef
79.
go back to reference Pleger ST. Cardiac AAV9-S100A1 gene therapy rescues post-ischemic heart failure in a preclinical large animal model. Sci Transl Med. 2011;3(92):92ra64.PubMedCrossRef Pleger ST. Cardiac AAV9-S100A1 gene therapy rescues post-­ischemic heart failure in a preclinical large animal model. Sci Transl Med. 2011;3(92):92ra64.PubMedCrossRef
80.
go back to reference Mikroulis D et al. Angiogenic growth factors in the treatment of peripheral arterial disease. Curr Vasc Pharmacol. 2007;5(3):195–209.PubMedCrossRef Mikroulis D et al. Angiogenic growth factors in the treatment of peripheral arterial disease. Curr Vasc Pharmacol. 2007;5(3):195–209.PubMedCrossRef
81.
go back to reference Henry TD et al. Safety of a non-viral plasmid-encoding dual isoforms of hepatocyte growth factor in critical limb ischemia patients: a phase I study. Gene Ther. 2011;18(8):788–94.PubMedCrossRef Henry TD et al. Safety of a non-viral plasmid-encoding dual isoforms of hepatocyte growth factor in critical limb ischemia patients: a phase I study. Gene Ther. 2011;18(8):788–94.PubMedCrossRef
82.
go back to reference Yang ZJ 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(8):790–6.PubMedCrossRef Yang ZJ 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(8):790–6.PubMedCrossRef
83.
go back to reference Kastrup J et al. Direct intramyocardial 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(7):982–8.PubMedCrossRef Kastrup J et al. Direct intramyocardial 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(7):982–8.PubMedCrossRef
84.
go back to reference Gyongyosi M et al. NOGA-guided analysis of regional myocardial perfusion abnormalities treated with intramyocardial injections of plasmid encoding vascular endothelial growth factor A-165 in patients with chronic myocardial ischemia: subanalysis of the EUROINJECT-ONE multicenter double-blind randomized study. Circulation. 2005;112(9 Suppl):I157–65.PubMed Gyongyosi M et al. NOGA-guided analysis of regional myocardial perfusion abnormalities treated with intramyocardial injections of plasmid encoding vascular endothelial growth factor A-165 in patients with chronic myocardial ischemia: subanalysis of the EUROINJECT-ONE multicenter double-blind randomized study. Circulation. 2005;112(9 Suppl):I157–65.PubMed
85.
go back to reference Stewart DJ 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(6):1109–15.PubMedCrossRef Stewart DJ 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(6):1109–15.PubMedCrossRef
86.
go back to reference Zou YR et al. Function of the chemokine receptor CXCR4 in haematopoiesis and in cerebellar development. Nature. 1998;393(6685):595–9.PubMedCrossRef Zou YR et al. Function of the chemokine receptor CXCR4 in haematopoiesis and in cerebellar development. Nature. 1998;393(6685):595–9.PubMedCrossRef
87.
go back to reference Askari AT et al. Effect of stromal-cell-derived factor 1 on stem-cell homing and tissue regeneration in ischaemic cardiomyopathy. Lancet. 2003;362(9385):697–703.PubMedCrossRef Askari AT et al. Effect of stromal-cell-derived factor 1 on stem-cell homing and tissue regeneration in ischaemic cardiomyopathy. Lancet. 2003;362(9385):697–703.PubMedCrossRef
88.
go back to reference Penn MS et al. Role of stem cell homing in myocardial regeneration. Int J Cardiol. 2004;95 Suppl 1:S23–5.PubMedCrossRef Penn MS et al. Role of stem cell homing in myocardial regeneration. Int J Cardiol. 2004;95 Suppl 1:S23–5.PubMedCrossRef
89.
go back to reference Zhang M et al. SDF-1 expression by mesenchymal stem cells results in trophic support of cardiac myocytes after myocardial infarction. FASEB J. 2007;21(12):3197–207.PubMedCrossRef Zhang M et al. SDF-1 expression by mesenchymal stem cells results in trophic support of cardiac myocytes after myocardial infarction. FASEB J. 2007;21(12):3197–207.PubMedCrossRef
90.
go back to reference Saxena A et al. Stromal cell-derived factor-1alpha is cardioprotective after myocardial infarction. Circulation. 2008;117(17):2224–31.PubMedCrossRef Saxena A et al. Stromal cell-derived factor-1alpha is cardioprotective after myocardial infarction. Circulation. 2008;117(17):2224–31.PubMedCrossRef
91.
go back to reference Tang J et al. Adenovirus-mediated stromal cell-derived factor-1 alpha gene transfer improves cardiac structure and function after experimental myocardial infarction through angiogenic and antifibrotic actions. Mol Biol Rep. 2010;37(4):1957–69.PubMedCrossRef Tang J et al. Adenovirus-mediated stromal cell-derived factor-1 alpha gene transfer improves cardiac structure and function after experimental myocardial infarction through angiogenic and antifibrotic actions. Mol Biol Rep. 2010;37(4):1957–69.PubMedCrossRef
92.
go back to reference Sundararaman S et al. Plasmid-based transient human stromal cell-derived factor-1 gene transfer improves cardiac function in chronic heart failure. Gene Ther. 2011;18(9):867–73.PubMedCrossRef Sundararaman S et al. Plasmid-based transient human stromal cell-derived factor-1 gene transfer improves cardiac function in chronic heart failure. Gene Ther. 2011;18(9):867–73.PubMedCrossRef
93.
go back to reference Pyo RT et al. CXCR4 modulates contractility in adult cardiac myocytes. J Mol Cell Cardiol. 2006;41(5):834–44.PubMedCrossRef Pyo RT et al. CXCR4 modulates contractility in adult cardiac myocytes. J Mol Cell Cardiol. 2006;41(5):834–44.PubMedCrossRef
94.
go back to reference LaRocca TJ. β2-Adrenergic receptor signaling in the cardiac myocyte is modulated by interactions with CXCR4. J Cardiovasc Pharmacol. 2010;56(5):548–59.PubMedCrossRef LaRocca TJ. β2-Adrenergic receptor signaling in the cardiac myocyte is modulated by interactions with CXCR4. J Cardiovasc Pharmacol. 2010;56(5):548–59.PubMedCrossRef
95.
go back to reference LaRocca TJ, Jeong D, Chen J, Kohlbrenner E, Lee A, Hallar RJ, et al. CXCR4 gene transfer prevents pressure overload induced heart failure. J Mol Cell Cardiol. 2012;53(2):223–32.PubMedCrossRef LaRocca TJ, Jeong D, Chen J, Kohlbrenner E, Lee A, Hallar RJ, et al. CXCR4 gene transfer prevents pressure overload induced heart failure. J Mol Cell Cardiol. 2012;53(2):223–32.PubMedCrossRef
go back to reference Asokan A, Schaffer DV, Samulski RJ. The AAV vector toolkit: poised at the clinical crossroads. Mol Ther. 2012;20(4):699–708.PubMedCrossRef Asokan A, Schaffer DV, Samulski RJ. The AAV vector toolkit: poised at the clinical crossroads. Mol Ther. 2012;20(4):699–708.PubMedCrossRef
go back to reference Kotin RM. Large-scale recombinant adeno-associated virus production. Hum Mol Genet. 2011 Apr 15;20(R1):R2–6.PubMedCrossRef Kotin RM. Large-scale recombinant adeno-associated virus production. Hum Mol Genet. 2011 Apr 15;20(R1):R2–6.PubMedCrossRef
go back to reference Rosas LE, et al. Patterns of scAAV vector insertion associated with oncogenic events in a mouse model for genotoxicity. Mol Ther. 2012 Nov;20(11):2098–110. doi:10.1038/mt.2012.197.PubMedCrossRef Rosas LE, et al. Patterns of scAAV vector insertion associated with oncogenic events in a mouse model for genotoxicity. Mol Ther. 2012 Nov;20(11):2098–110. doi:10.1038/mt.2012.197.PubMedCrossRef
go back to reference Merlet E, et al. A calcium-sensitive promoter construct for gene therapy. Gene Ther. 2012 Mar 29. doi:10.1038/gt.2012.30.PubMedCrossRef Merlet E, et al. A calcium-sensitive promoter construct for gene therapy. Gene Ther. 2012 Mar 29. doi:10.1038/gt.2012.30.PubMedCrossRef
Metagegevens
Titel
Cardiovascular Gene Therapy
Auteurs
Thomas J. LaRocca, MD, PhD
Roger J. Hajjar, MD
Copyright
2013
Uitgeverij
Springer New York
DOI
https://doi.org/10.1007/978-1-4614-6705-2_44