Abstract
The expression of recombinant genes in the human coronary arteries and myocardium holds promise for the treatment of a number of inherited and acquired cardiovascular diseases. These include the cardiomyopathy associated with Duchenne Muscular Dystrophy (DMD) (For review, Perloff, 1992), the problem of restenosis following balloon angioplasty of the coronary arteries (For review, Safian et al., 1992) and syndromes of chronic myocardial ischemia (For review, Rutherford and Braunwald, 1992). Current approaches to somatic gene therapy can be divided into two general categories: Ex vivo gene transfer involves the removal of cells from an organism followed by gene transduction in vitro and reimplantation of the genetically modified cells into the appropriate tissue in vivo. In contrast, in vivo gene transfer involves the introduction of a recombinant gene into the appropriate cell type in vivo without the need to remove and culture cells from the recipient organism.
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References
Acsadi G, Dickson G, Love DR, Jani A, Walsh FS, Gurusinghe A, Wolff JA, Davies KE (1991): Human dystrophin expression in mdx mice after intra-muscular injection of DNA constructs. Nature 352: 815–818
Acsadi G, Jiao S, Jani A, Duke D, Williams P, Chong W, Wolff JA (1991): Direct gene transfer and expression into rat heart in vivo. New Biol 3: 71–81
Barr E, Carroll J, Tripathy S, Kozarsky K, Wilson JM, Leiden JM (1993): Percutaneous transluminal gene transfer into the heart using replication-defective recombinant adenovirus. (Submitted.)
Berkner KL (1988): Development of adenovirus vectors for the expression of heterologous genes. Biotechniques 6: 616–629
Buttrick PM, Kass A, Kitsis RN, Kaplan ML, Leinwand LA (1992): Behavior of genes directly injected into the rat heart in vivo. Circ Res 70: 193–198
Chamley JH, Campbell GR, McConnell, JD, Groschel-Stewart U (1977): Comparison of vascular smooth muscle cells from adult human, monkey, and rabbit in primary culture and in subculture. Cell Tiss Res 111: 503–522
Chanock RM, Ludwig W, Heubner RJ, Cate TR, Chu LW (1966): Immunization by selective infection with type 4 adenovirus grown in human diploid tissue cultures. I. Safety and lack of oncogenicity and tests for potency in volunteers. JAMA 195: 445–452
Dai Y, Roman M, Naviaux RK, Verma I (1992): Gene therapy via primary myoblasts: Long-term expression of factor IX protein following transplantation in vivo. Proc Natl Acad Sci USA 89: 10892–10895
Flugelman MY, Jaklitsch MT, Newman KD, Casscells W, Bratthauer GL, Dichek DA (1992): Low level in vivo gene transfer into the arterial wall thromgh a perforated balloon catheter. Circulation 85: 1110–1117
Gal D, Weir L, LeClerc G, Pickering JG, Hogan J, Isner JM (1993): Direct myocardial transfection in two animal models. Evaluation of parameters affecting gene expression and percutaneous gene delivery. Lab Invest 68: 18–25
Graham FL, Eb AJ-van-der (1973): Transformation of rat cells by DNA of human adenovirus 5. Virology 54: 536–539
Graham FL, Smiley J, Russell WC, Nairn R (1977): Characteristics of a human cell line transformed by DNA from adenovirus type 5. J Gen Virol 36: 59–74
Gustafson T, Markham B, Bahl JJ, Morkin E (1987): Thyroid hormone regulates expression of a transfected a myosin heavy chain fusion gene in fetal heart cells. Proc Natl Acad Sci USA 84: 3122–3126
Haj-Ahmad Y, Graham FL (1986): Development of a helper-independent human adenovirus vector and its use in the transfer of the herpes simplex virus thymidine kinase gene. J Virol 57: 267–274
Horwitz MS (1990): The Adenoviruses. In Virology, Fields BN, Knipe DM, eds. New York: Raven Press
Izumo S, Nadal-Ginard B, Mahdavi V (1986): All members of the MHC multigene family respond to thyroid hormone in a highly tissue-specific manner. Science 231: 597–600
Jiao S, Williams P, Berg RK, Hodgeman BA, Liu L, Repetto G, Wolff JA (1992): Direct gene transfer into nonhuman primate myofibers in vivo. Hum Gene Ther 3: 21–33
Kitsis R, Buttrick P, McNally E, Kaplan M, Leinwand LA (1991): Hormonal modulation of a gene injected into rat heart in vivo. Proc Natl Acad Sci USA 88: 4138–4142
Le Gal La Salle G, Robert JJ, Berrard RS, Ridoux V, Stratford-Perricaudet LD, Perricaudet M, Mallet J (1993): An adenovirus vector for gene transfer into neurons and glia in the brain. Science 259: 988–990
Leinwand LA, Leiden JM (1991): Gene transfer into cardiac myocytes in vivo. Trends Card Med 1: 271–276
Lin H, Parmacek MS, Morie G, Boiling S, Leiden JM (1990): Expression of recombinant genes in myocardium in vivo after direct injection of DNA. Circulation 82: 2217–2221
Lompre A-M, Nadal-Ginard B, Mahdavi V (1984): Expression of the cardiac ventricular a- and b-myosin heavy chain genes is developmentally and hormon- ally regulated. J Biol Chem 259: 6437–6446
Muller DW, Ellis SG, Topol EJ (1992): Experimental models of coronary artery restenosis. J Am Coll Cardiol 19: 418–432
Nabel EG, Plautz G, Boyce FM, Stanley JC, Nabel GJ (1989): Recombinant gene expression in vivo within endothelial cells of the arterial wall. Science 244: 1342–1344
Nabel EG, Plautz G, Nabel GJ (1990): Site-specific gene expression in vivo by direct gene transfer into the arterial wall. Science 249: 1285–1288
Nabel EG, Plautz G, Nabel GJ (1991): Gene transfer into vascular cells. J Am Coll Cardiol 17: 189B–194B
Overbeek PA, Lai S-P, Van Quill KR, Westphal H (1986): Tissue-specific expression in transgenic mice of a fused gene containing RSV terminal sequences. Science 231: 1574–1577
Parmacek MS, Vora AJ, Shen T, Barr E, Jung F, Leiden JM (1992): Identification and characterization of a cardiac-specific transcriptional regultory element in the slow/cardiac troponin C gene. Mol Cell Biol 12: 1967–1976
Perloff JK (1992): Congenital heart disease in adults. In: Heart Disease, Braunwald E, ed. Philadelphia: Saunders Press
Quantin B, Perricaudet LD, Tajbakhsh S, Mandel J-L (1992): Adenovirus as an expression vector in muscle cells in vivo. Proc Natl Acad Sci USA 89: 2581–2584
Ragot T, Vincent N, Chafey P, Vigne E, Gilgenkrantz H, et al. (1993): Efficient adenovirus-mediated transfer of a human minidystrophin gene to skeletal muscle of mdx mice. Nature 361: 647–650
Rosenfeld MA, Siegfried W, Yoshimura K, Yoneyama K, et al. (1991): Adenovirus-mediated transfer of a recombinant al-antitrypsin gene to the lung epithelium in vivo. Science 252: 431–434
Rosenfeld MA, Yoshimura K, Trapnell BC, Yonyama K, et al. (1992): In vivo transfer of the human cystic fibrosis transmembrane conductance regulator gene to the airway epithelium. Cell 68: 143–155
Rutherford JD, Braunwald E (1992): Chronic ischemic heart disease. In: Heart Disease, Braunwald E, ed. Philadelphia: Saunders Press
Sabia PJ, Powers ER, Ragosta M, Sarembock IJ, Burwell LR, Kaul S (1992): An association between collateral blood flow and myocardial viability in patients with recent myocardial infarction. N Eng J Med 327: 1825–1831
Safian RD, Gelbfish JS, Erny RE, Schnitt SJ, Schmidt DA, Baim DS (1990): Coronary atherectomy. Clinical, angiographic, and histological findings and observations regarding potential mechanims. Circulation 82: 69–79
Simons M, Edelman ER, DeKeyser J-LL, Langer R, Rosenberg RD (1992): Antisense c-myb oligonucleotides inhibit intimal arterial smooth muscle cell accumulation in vivo. Nature 359: 67–70
Simons M, Rosenberg RD (1992): Antisense nonmuscle myosin heaavy chain and c-myb oligonucleotides suppress smooth muscle cell proliferation in vitro. Cm Res 70: 835–843
Steinhelper ME, Lanson NA, Dresdner KP, et al. (1990): Proliferation in vivo and in culture of differentiated adult atrial cardiomyocytes from transgenic mice. Am J Physiol 259: H1826–H1834
Stratford-Pericaudet LD, Makeh I, Perricaudet M, Briand P (1992): Widespread long-term gene transfer to mouse skeletal muscles and heart. J Clin Invest 90: 626–630
Swain JL (1989): Gene therapy: a new approach to the treatment of cardiovascular disease. Circulation 80: 1495–1496
Tsika R, Bahl J, Leinwand L, Morkin E (1990): Thyroid hormone regulates expression of a transfected human a-myosin heavy chain fusion gene in fetal rat heart cells. Proc Natl Acad Sei USA 87: 379–383
Watanabe AM, Green FJ, Farmer BB (1986): Preparation and use of cardiac myocytes in experimental cardiology. In. The Heart and Cardiovascular System. Fozzard HA, Haber E, Jennings RB, Katz AM, Morgan, HE, eds. New York: Raven Press
Wolff JA, Malone RW, Williams P, et al. (1990): Direct gene transfer into mouse muscle in vivo. Science 247: 1465–1468
Wolff JA, Williams P, Acsadi G, Jiao S, Jani A, Chong W (1991): Conditions affecting direct gene transfer into rodent muscle in vivo. BioTechniques 11: 474–485
Wu CH, Wilson JM, Wu GY (1989): Targeting genes: delivery and persistent expression of a foreign gene driven by mammalian regulatory elements in vivo. J Biol Chem 264: 16985–16987
Zak R (1974): Development and proliferation capacity of cardiac muscle cells. Circ Res 34–35 (suppl II): II–17
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© 1994 Birkhäuser Boston
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Leiden, J.M., Barr, E. (1994). In Vivo Gene Transfer into the Heart. In: Wolff, J.A. (eds) Gene Therapeutics. Birkhäuser Boston. https://doi.org/10.1007/978-1-4684-6822-9_20
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DOI: https://doi.org/10.1007/978-1-4684-6822-9_20
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