Prospects for gene therapy for inherited cardiomyopathies

Manolio, 1992, Prevalence and etiology of idiopathic dilated cardiomyopathy (summary of a National Heart, Lung, and Blood Institute workshop), Am J Cardiol, 69, 1458, 10.1016/0002-9149(92)90901-A Kasper, 1994, The causes of dilated cardiomyopathy: a clinicopathologic review of 673 consecutive patients, J Am Coll Cardiol, 23, 586, 10.1016/0735-1097(94)90740-4 Dec, 1994, Idiopathic dilated cardiomyopathy, New Engl J Med, 331, 1564, 10.1056/NEJM199412083312307 Kelly, 1994, Inherited cardiomyopathies, New Engl J Med, 330, 913, 10.1056/NEJM199403313301308 Michels, 1992, The frequency of familial dilated cardiomyopathy in a series of patients with idiopathic dilated cardiomyopathy, New Engl J Med, 326, 77, 10.1056/NEJM199201093260201 Berko, 1987, X-Linked dilated cardiomyopathy, New Engl J Med, 316, 1186, 10.1056/NEJM198705073161904 Muntoni, 1993, Brief report: deletion of the dystrophin muscle-promoter region associated with X-linked dilated cardiomyopathy [see comments], New Engl J Med, 329, 921, 10.1056/NEJM199309233291304 Muntoni, 1995, A mutation in the dystrophin gene selectively affecting dystrophin expression in the heart, J Clin Invest, 96, 693, 10.1172/JCI118112 Ortiz-Lopez, 1997, Evidence for a dystrophin missense mutation as a cause of X-linked dilated cardiomyopathy, Circulation, 95, 2434, 10.1161/01.CIR.95.10.2434 Klietsch, 1993, Dystrophin-glycoprotein complex and laminin colocalize to the sarcolemma and transverse tubules of cardiac muscle, Circ Res, 72, 349, 10.1161/01.RES.72.2.349 Koenig, 1988, The complete sequence of dystrophin predicts a rod-shaped cytoskeletal protein, Cell, 53, 219, 10.1016/0092-8674(88)90383-2 Koenig, 1990, Detailed analysis of the repeat domain of dystrophin reveals four potential hinge segments that may confer flexibility, J Biol Chem, 265, 4560, 10.1016/S0021-9258(19)39599-7 Ohlendieck, 1996, Towards an understanding of the dystrophin-glycoprotein complex: linkage between the extracellular matrix and the membrane cytoskeleton in muscle fibers, Eur J Cell Biol, 69, 1 Hoffman, 1987, Dystrophin: the protein product of the Duchenne muscular dystrophy locus, Cell, 51, 919, 10.1016/0092-8674(87)90579-4 Towbin, 1993, X-Linked dilated cardiomyopathy. Molecular genetic evidence of linkage to the Duchenne muscular dystrophy (dystrophin) gene at the Xp21 locus, Circulation, 87, 1854, 10.1161/01.CIR.87.6.1854 Nigro, 1994, Mutation of dystrophin gene and cardiomyopathy, Neuromusc Disord, 4, 371, 10.1016/0960-8966(94)90073-6 Nigro, 1996, Autosomal recessive limb-girdle muscular dystrophy, LGMD2F, is caused by a mutation in the delta-sarcoglycan gene, Nat Genet, 14, 195, 10.1038/ng1096-195 Nigro, 1997, Identification of the Syrian hamster cardiomyopathy gene, Hum Mol Genet, 6, 601, 10.1093/hmg/6.4.601 Sakamoto, 1997, Both hypertrophic and dilated cardiomyopathies are caused by mutation of the same gene, delta-sarcoglycan, in hamster: an animal model of disrupted dystrophin-associated glycoprotein complex, Proc Natl Acad Sci USA, 94, 13873, 10.1073/pnas.94.25.13873 Arber, 1997, MLP-deficient mice exhibit a disruption of cardiac cytoarchitectural organization, dilated cardiomyopathy, and heart failure, Cell, 88, 393, 10.1016/S0092-8674(00)81878-4 Maeda, 1997, Dilated cardiomyopathy associated with deficiency of the cytoskeletal protein metavinculin, Circulation, 95, 17, 10.1161/01.CIR.95.1.17 Sakamoto, 1999, Delineation of genomic deletion in cardiomyopathic hamster, FEBS Lett, 447, 124, 10.1016/S0014-5793(99)00267-7 Bione, 1996, A novel X-linked gene, G4.5, is responsible for Barth syndrome, Nat Genet, 12, 385, 10.1038/ng0496-385 Barth, 1983, An X-linked mitochondrial disease affecting cardiac muscle, skeletal muscle and neutrophil leucocytes, J Neurol Sci, 62, 327, 10.1016/0022-510X(83)90209-5 Bleyl, 1997, Neonatal, lethal noncompaction of the left ventricular myocardium is allelic with Barth syndrome, Am J Hum Genet, 61, 868, 10.1086/514879 Bione, 1994, Identification of a novel X-linked gene responsible for Emery-Dreifuss muscular dystrophy, Nat Genet, 8, 323, 10.1038/ng1294-323 Durand, 1995, Localization of a gene responsible for familial dilated cardiomyopathy to chromosome 1q32, Circulation, 92, 3387, 10.1161/01.CIR.92.12.3387 Siu, 1999, Familial dilated cardiomyopathy locus maps to chromosome 2q31, Circulation, 99, 1022, 10.1161/01.CIR.99.8.1022 Krajinovic, 1995, Linkage of familial dilated cardiomyopathy to chromosome 9. Heart Muscle Disease Study Group, Am J Hum Genet, 57, 846 Bowles, 1996, Gene mapping of familial autosomal dominant dilated cardiomyopathy to chromosome 10q21-23, J Clin Invest, 98, 1355, 10.1172/JCI118922 Olson, 1998, Actin mutations in dilated cardiomyopathy, a heritable form of heart failure, Science, 280, 750, 10.1126/science.280.5364.750 Kass, 1994, A gene defect that causes conduction system disease and dilated cardiomyopathy maps to chromosome 1p1-1q1, Nat Genet, 7, 546, 10.1038/ng0894-546 Jung, 1999, Investigation of a family with autosomal dominant dilated cardiomyopathy defines a novel locus on chromosome 2q14-q22, Am J Hum Genet, 65, 1068, 10.1086/302580 Olson, 1996, Mapping a cardiomyopathy locus to chromosome 3p22-p25, J Clin Invest, 97, 528, 10.1172/JCI118445 Messina, 1997, Linkage of familial dilated cardiomyopathy with conduction defect and muscular dystrophy to chromosome 6q23, Am J Hum Genet, 61, 909, 10.1086/514896 Bonne, 1999, Mutations in the gene encoding lamin A/C cause autosomal dominant Emery-Dreifuss muscular dystrophy, Nat Genet, 21, 285, 10.1038/6799 Salmikangas, 1999, Myotilin, a novel sarcomeric protein with two Ig-like domains, is encoded by a candidate gene for limb-girdle muscular dystrophy, Hum Mol Genet, 8, 1329, 10.1093/hmg/8.7.1329 Li, 1999, Desmin mutation responsible for idiopathic dilated cardiomyopathy, Circulation, 100, 461, 10.1161/01.CIR.100.5.461 Thornell, 1997, Null mutation in the desmin gene gives rise to a cardiomyopathy, J Mol Cell Cardiol, 29, 2107, 10.1006/jmcc.1997.0446 Carlson, 1998, The dystrophinopathies: an alternative to the structural hypothesis, Neurobiol Dis, 5, 3, 10.1006/nbdi.1998.0188 Bowles, 1986, Detection of Coxsackie-B-virus-specific RNA sequences in myocardial biopsy samples from patients with myocarditis and dilated cardiomyopathy, Lancet, 1, 1120, 10.1016/S0140-6736(86)91837-4 Kandolf, 1987, In situ detection of enteroviral genomes; in myocardial cells by nucleic acid hybridization: an approach to the diagnosis of viral heart disease, Proc Natl Acad Sci USA, 84, 6272, 10.1073/pnas.84.17.6272 Bowles, 1989, End-stage dilated cardiomyopathy. Persistence of enterovirus RNA in myocardium at cardiac transplantation and lack of immune response, Circulation, 80, 1128, 10.1161/01.CIR.80.5.1128 Martin, 1994, Acute myocarditis: rapid diagnosis by PCR in children, Circulation, 90, 330, 10.1161/01.CIR.90.1.330 Griffin, 1995, Analysis of formalin-fixed and frozen myocardial autopsy samples for viral genome in childhood myocarditis and dilated cardiomyopathy with endocardial fibroelastosis using polymerase chain reaction (PCR), Cardiovascular Pathol, 4, 3, 10.1016/1054-8807(94)00025-M Pauschinger, 1999, Detection of adenoviral genome in the myocardium of adult patients with idiopathic left ventricular dysfunction, Circulation, 99, 1348, 10.1161/01.CIR.99.10.1348 Prince, 1993, Pathogenesis of adenovirus type 5 pneumonia in cotton rats (Sigmodon hispidus), J Virol, 67, 101, 10.1128/JVI.67.1.101-111.1993 Bowles, 1999, An animal model of adenovirus-induced myocarditis, Pediatr Res, 45, 20, 10.1203/00006450-199904020-00124 Cunningham, 1990, Persistence of enteroviral RNA in chronic fatigue syndrome is associated with the abnormal production of equal amounts of positive and negative strands of enteroviral RNA, J Gen Virol, 71, 1399, 10.1099/0022-1317-71-6-1399 Why, 1994, Clinical and prognostic significance of detection of enteroviral RNA in the myocardium of patients with myocarditis or dilated cardiomyopathy, Circulation, 89, 2582, 10.1161/01.CIR.89.6.2582 Badorff, 1999, Enteroviral protease 2A cleaves dystrophin: evidence of cytoskeletal disruption in an acquired cardiomyopathy, Nat Med, 5, 320, 10.1038/6543 Krajcer, 1988, Mitral valve replacement and septal myomectomy in hypertrophic cardiomyopathy. Ten-year follow-up in 80 patients, Circulation, 78, I35 Fananapazir, 1994, Long-term results of dual-chamber (DDD) pacing in obstructive hypertrophic cardiomyopathy. Evidence for progressive symptomatic and hemodynamic improvement and reduction of left ventricular hypertrophy, Circulation, 90, 2731, 10.1161/01.CIR.90.6.2731 Geisterfer-Lowrance, 1990, A molecular basis for familial hypertrophic cardiomyopathy: a beta cardiac myosin heavy chain gene missense mutation, Cell, 62, 999, 10.1016/0092-8674(90)90274-I Thierfelder, 1994, Alpha-tropomyosin and cardiac troponin T mutations cause familial hypertrophic cardiomyopathy: a disease of the sarcomere, Cell, 77, 701, 10.1016/0092-8674(94)90054-X Kimura, 1997, Mutations in the cardiac troponin I gene associated with hypertrophic cardiomyopathy, Nat Genet, 16, 379, 10.1038/ng0897-379 Bonne, 1995, Cardiac myosin binding protein-C gene splice acceptor site mutation is associated with familial hypertrophic cardiomyopathy, Nat Genet, 11, 438, 10.1038/ng1295-438 Watkins, 1995, Mutations in the cardiac myosin binding protein-C gene on chromosome 11 cause familial hypertrophic cardiomyopathy, Nat Genet, 11, 434, 10.1038/ng1295-434 Poetter, 1996, Mutations in either the essential or regulatory light chains of myosin are associated with a rare myopathy in human heart and skeletal muscle, Nat Genet, 13, 63, 10.1038/ng0596-63 Mogensen, 1999, Alpha-cardiac actin is a novel disease gene in familial hypertrophic cardiomyopathy, J Clin Invest, 103, R39, 10.1172/JCI6460 Rayment, 1993, Structure of the actin-myosin complex and its implications for muscle contraction, Science, 261, 58, 10.1126/science.8316858 Towbin, 1998, The role of cytoskeletal proteins in cardiomyopathies, Curr Opin Cell Biol, 10, 131, 10.1016/S0955-0674(98)80096-3 Towbin, 1999, Etiologies of cardiomyopathy and heart failure, Nat Med, 5, 266, 10.1038/6474 Crystal, 1995, The gene as the drug, Nat Med, 1, 15, 10.1038/nm0195-15 Nabel, 1995, Gene therapy for vascular diseases, Atherosclerosis, 118, S51, 10.1016/0021-9150(95)90073-X Wilson, 1996, Adenoviruses as gene-delivery vehicles, New Engl J Med, 334, 1185, 10.1056/NEJM199605023341809 Marshall, 1998, Recent advances in skeletal-muscle-based gene therapy, Curr Opin Genet Dev, 8, 360, 10.1016/S0959-437X(98)80094-4 Kay, 1999, Gene therapy for the hemophilias, Proc Natl Acad Sci USA, 96, 9973, 10.1073/pnas.96.18.9973 Losordo, 1999, Gene therapy for myocardial angiogenesis, Am Heart J, 138, 132, 10.1016/S0002-8703(99)70333-9 Kass-Eisler, 1994, The impact of developmental stage, route of administration and the immune system on adenovirus-mediated gene transfer, Gene Ther, 1, 395 Gilgenkrantz, 1995, Transient expression of genes transferred in vivo into heart using first-generation adenoviral vectors: role of the immune response, Hum Gene Ther, 6, 1265, 10.1089/hum.1995.6.10-1265 Wang, 1996, Adenovirus-mediated gene transfer into rat cardiac allografts. Comparison of direct injection and perfusion, Transplantation, 61, 1726, 10.1097/00007890-199606270-00011 Lee, 1996, Cardiac gene transfer by intracoronary infusion of adenovirus vector-mediated reporter gene in the transplanted mouse heart, J Thorac Cardiovasc Surg, 111, 246, 10.1016/S0022-5223(96)70422-1 Magovern CJ, Mack CA, Zhang J et al. Direct in vivo gene transfer to canine myocardium using a replication-deficient adenovirus vector. Ann Thorac Surg 1996;62:425–433; discussion: 433–434. French, 1994, Direct in vivo gene transfer into porcine myocardium using replication-deficient adenoviral vectors, Circulation, 90, 2414, 10.1161/01.CIR.90.5.2414 Barr, 1994, Efficient catheter-mediated gene transfer into the heart using replication-defective adenovirus, Gene Ther, 1, 51 Stratford-Perricaudet, 1992, Widespread long-term gene transfer to mouse skeletal muscles and heart, J Clin Invest, 90, 626, 10.1172/JCI115902 Huard, 1995, The route of administration is a major determinant of the transduction efficiency of rat tissues by adenoviral recombinants, Gene Ther, 2, 107 Donahue, 1998, Acceleration of widespread adenoviral gene transfer to intact rabbit hearts by coronary perfusion with low calcium and serotonin, Gene Ther, 5, 630, 10.1038/sj.gt.3300649 Woo, 1997, In utero cardiac gene transfer via intraplacental delivery of recombinant adenovirus, Circulation, 96, 3561, 10.1161/01.CIR.96.10.3561 Yang, 1995, Cellular and humoral immune responses to viral antigens create barriers to lung-directed gene therapy with recombinant adenoviruses, J Virol, 69, 2004, 10.1128/JVI.69.4.2004-2015.1995 Fang, 1996, Lack of persistence of E1-recombinant adenoviral vectors containing a temperature-sensitive E2A mutation in immunocompetent mice and hemophilia B dogs, Gene Ther, 3, 217 Engelhardt, 1994, Prolonged transgene expression in cotton rat lung with recombinant adenoviruses defective in E2a, Hum Gene Ther, 5, 1217, 10.1089/hum.1994.5.10-1217 Lee, 1995, The constitutive expression of the inununomodulatory gp19k protein in E1-, E3-adenoviral vectors strongly reduces the host cytotoxic T cell response against the vector, Gene Ther, 2, 256 Gao, 1996, Biology of adenovirus vectors with E1 and E4 deletions for liver-directed gene therapy, J Virol, 70, 8934, 10.1128/JVI.70.12.8934-8943.1996 Wold, 1989, Adenovirus region E3 proteins that prevent cytolysis by cytotoxic T cells and tumor necrosis factor, Mol Biol Med, 6, 433 Fisher, 1996, Recombinant adenovirus deleted of all viral genes for gene therapy of cystic fibrosis, Virology, 217, 11, 10.1006/viro.1996.0088 Kochanek, 1996, A new adenoviral vector: replacement of all viral coding sequences with 28 kb of DNA independently expressing both full-length dystrophin and beta-galactosidase, Proc Natl Acad Sci USA, 93, 5731, 10.1073/pnas.93.12.5731 Chen, 1999, DNA from both high-capacity and first-generation adenoviral vectors remains intact in skeletal muscle, Hum Gene Ther, 10, 365, 10.1089/10430349950018814 Chen, 1997, Persistence in muscle of an adenoviral vector that lacks all viral genes, Proc Natl Acad Sci USA, 94, 1645, 10.1073/pnas.94.5.1645 Clemens, 1996, In vivo muscle gene transfer of full-length dystrophin with an adenoviral vector that lacks all viral genes, Gene Ther, 3, 965 Barr, 1995, Strain related variations in adenovirally mediated transgene expression from mouse hepatocytes in vivo: comparisons between immunocompetent and immunodeficient inbred strains, Gene Ther, 2, 151 Zepeda, 1996, Neonatal cotton rats do not exhibit destructive immune responses to adenoviral vectors, Gene Ther, 3, 973 Fang, 1995, Gene therapy for hemophilia B: host immunosuppression prolongs the therapeutic effect of adenovirus-mediated factor IX expression, Hum Gene Ther, 6, 1039, 10.1089/hum.1995.6.8-1039 Kay, 1995, Long-term hepatic adenovirus-mediated gene expression in mice following CTLA4Ig administration, Nat Genet, 11, 191, 10.1038/ng1095-191 Bergelson, 1997, Isolation of a common receptor for Coxsackie B viruses and adenoviruses 2 and 5, Science, 275, 1320, 10.1126/science.275.5304.1320 Tomko, 1997, HCAR and MCAR: the human and mouse cellular receptors for subgroup C adenoviruses and group B coxsackie viruses, Proc Natl Acad Sci USA, 94, 3352, 10.1073/pnas.94.7.3352 Lee, 1992, Myosin light chain-2 luciferase transgenic mice reveal distinct regulatory programs for cardiac and skeletal muscle-specific expression of a single contractile protein gene, J Biol Chem, 267, 15875, 10.1016/S0021-9258(19)49616-6 Rothmann, 1996, Heart muscle-specific gene expression using replication defective recombinant adenovirus, Gene Ther, 3, 919 Griscelli, 1998, Heart-specific targeting of beta-galactosidase by the ventricle-specific cardiac myosin light chain 2 promoter using adenovirus vectors, Hum Gene Ther, 9, 1919, 10.1089/hum.1998.9.13-1919 Wang, 1994, A regulatory system for use in gene transfer, Proc Natl Acad Sci USA, 91, 8180, 10.1073/pnas.91.17.8180 Baron, 1995, Co-regulation of two gene activities by tetracycline via a bidirectional promoter, Nucleic Acids Res, 23, 3605, 10.1093/nar/23.17.3605 Dhawan, 1995, Tetracycline-regulated gene expression following direct gene transfer into mouse skeletal muscle, Somat Cell Mol Genet, 21, 233, 10.1007/BF02255778 Qin, 1995, Multiple vectors effectively achieve gene transfer in a murine cardiac transplantation model. Immunosuppression with TGF-beta 1 or vIL-10, Transplantation, 59, 809, 10.1097/00007890-199503000-00002 Gojo, 1996, Ex vivo gene transfer into myocardium using replication-defective retrovirus, Cell Transplant, 5, S81, 10.1016/0963-6897(96)00047-4 Kay, 1992, Hepatic gene therapy: persistent expression of human alpha 1-antitrypsin in mice after direct gene delivery in vivo, Hum Gene Ther, 3, 641, 10.1089/hum.1992.3.6-641 Prentice, 1996, Ischemic/reperfused myocardium can express recombinant protein following direct DNA or retroviral injection, J Mol Cell Cardiol, 28, 133, 10.1006/jmcc.1996.0013 Kosai, 1998, Retrovirus-mediated in vivo gene transfer in the replicating liver using recombinant hepatocyte growth factor without liver injury or partial hepatectomy, Hum Gene Ther, 9, 1293, 10.1089/hum.1998.9.9-1293 Douar, 1997, Foetal gene delivery in mice by intra-amniotic administration of retroviral producer cells and adenovirus, Gene Ther, 4, 883, 10.1038/sj.gt.3300498 Kafri, 1997, Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors, Nat Genet, 17, 314, 10.1038/ng1197-314 Iwakuma, 1999, Self-inactivating lentiviral vectors with U3 and U5 modifications, Virology, 261, 120, 10.1006/viro.1999.9850 Takahashi, 1999, Rescue from photoreceptor degeneration in the rd mouse by human immunodeficiency virus vector-mediated gene transfer, J Virol, 73, 7812, 10.1128/JVI.73.9.7812-7816.1999 Rebolledo, 1998, Infection of human fetal cardiac myocytes by a human immunodeficiency virus-1-derived vector, Circ Res, 83, 738, 10.1161/01.RES.83.7.738 Matsushita, 1998, Adeno-associated virus vectors can be efficiently produced without helper virus, Gene Ther, 5, 938, 10.1038/sj.gt.3300680 Clark, 1999, Highly purified recombinant adeno-associated virus vectors are biologically active and free of detectable helper and wild-type viruses, Hum Gene Ther, 10, 1031, 10.1089/10430349950018427 Gao, 1998, High-titer adeno-associated viral vectors from a Rep/Cap cell line and hybrid shuttle virus, Hum Gene Ther, 9, 2353, 10.1089/hum.1998.9.16-2353 Recchia, 1999, Site-specific integration mediated by a hybrid adenovirus/adeno-associated virus vector, Proc Natl Acad Sci USA, 96, 2615, 10.1073/pnas.96.6.2615 Lieber, 1999, Integrating adenovirus-adeno-associated virus hybrid vectors devoid of all viral genes, J Virol, 73, 9314, 10.1128/JVI.73.11.9314-9324.1999 Maeda, 1998, Efficient gene transfer into cardiac myocytes using adeno-associated virus (AAV) vectors, J Mot Cell Cardiol, 30, 1341, 10.1006/jmcc.1998.0697 Svensson, 1999, Efficient and stable transduction of cardiomyocytes after intramyocardial injection or intracoronary perfusion with recombinant adeno-associated virus vectors, Circulation, 99, 201, 10.1161/01.CIR.99.2.201 Sawa, 1998, Efficient transfer of oligonucleotides and plasmid DNA into the whole heart through the coronary artery, Gene Ther, 5, 1472, 10.1038/sj.gt.3300750 Losordo, 1998, Gene therapy for myocardial angiogenesis: initial clinical results with direct myocardial injection of phVEGF165 as sole therapy for myocardial ischemia, Circulation, 98, 2800, 10.1161/01.CIR.98.25.2800 Partridge, 1989, Conversion of mdx myofibres from dystrophin-negative to -positive by injection of normal myoblasts, Nature, 337, 176, 10.1038/337176a0 England, 1990, Very mild muscular dystrophy associated with the deletion of 46% of dystrophin, Nature, 343, 180, 10.1038/343180a0 Wells, 1992, Human dystrophin expression corrects the myopathic phenotype in transgenic mdx mice, Hum Mol Genet, 1, 35, 10.1093/hmg/1.1.35 Ragot, 1993, Efficient adenovirus-mediated transfer of a human minidystrophin gene to skeletal muscle of mdx mice, Nature, 361, 647, 10.1038/361647a0 Vincent, 1993, Long-term correction of mouse dystrophic degeneration by adenovirus-mediated transfer of a minidystrophin gene, Nat Genet, 5, 130, 10.1038/ng1093-130 Alameddine, 1994, Expression of a recombinant dystrophin in mdx mice using adenovirus vector, Neuromuscul Disord, 4, 193, 10.1016/0960-8966(94)90020-5 Haecker, 1996, In vivo expression of full-length human dystrophin from adenoviral vectors deleted of all viral genes, Hum Gene Ther, 7, 1907, 10.1089/hum.1996.7.15-1907 Bouri, 1999, Polylysine modification of adenoviral fiber protein enhances muscle cell transduction, Hum Gene Ther, 10, 1633, 10.1089/10430349950017635 Feero, 1997, Viral gene delivery to skeletal muscle: insights on maturation-dependent loss of fiber infectivity for adenovirus and herpes simplex: type 1 viral vectors, Hum Gene Ther, 8, 371, 10.1089/hum.1997.8.4-371 Gilbert, 1998, Efficient utrophin expression following adenovirus gene transfer in dystrophic muscle, Biochem Biophys Res Commun, 242, 244, 10.1006/bbrc.1997.7936 Sharp, 1992, An error in dystrophin mRNA processing in golden retriever muscular dystrophy, an animal homologue of Duchenne muscular dystrophy, Genomics, 13, 115, 10.1016/0888-7543(92)90210-J Howell, 1998, High-level dystrophin expression after adenovirus-mediated dystrophin minigene transfer to skeletal muscle of dystrophic dogs: prolongation of expression with immunosuppression, Hum Gene Ther, 9, 629, 10.1089/hum.1998.9.5-629 Dunckley, 1992, Retroviral-mediated transfer of a dystrophin minigene into mdx mouse myoblasts in vitro, FEBS Lett, 296, 128, 10.1016/0014-5793(92)80363-L Fassati, 1997, Genetic correction of dystrophin deficiency and skeletal muscle remodeling in adult MDX mouse via transplantation of retroviral producer cells, J Clin Invest, 100, 620, 10.1172/JCI119573 Li, 1999, rAAV vector-mediated sarcogylcan gene transfer in a hamster model for limb girdle muscular dystrophy, Gene Ther, 6, 74, 10.1038/sj.gt.3300830 Greelish, 1999, Stable restoration of the sarcoglycan complex in dystrophic muscle perfused with histamine and a recombinant adeno-associated viral vector, Nat Med, 5, 439, 10.1038/7439 Marian, 1997, Expression of a mutant (Arg92Gln) human cardiac troponin T, known to cause hypertrophic cardiomyopathy, impairs adult cardiac myocyte contractility, Circ Res, 81, 76, 10.1161/01.RES.81.1.76 Tsukamoto, 1999, Enhanced expression of recombinant dystrophin following intramuscular injection of Epstein–Barr virus (EBV)-based mini-chromosome vectors in mdx mice, Gene Ther, 6, 1331, 10.1038/sj.gt.3300944 Huard, 1997, Gene transfer to muscle using herpes simplex virus-based vectors, Neuromuscul Disord, 7, 299, 10.1016/S0960-8966(97)00054-0 Yu, 1994, Progress towards gene therapy for HIV infection, Gene Ther, 1, 13 Bashkin, 1995, Ribozyme mimics as catalytic antisense reagents, Appl Biochem Biotechnol, 54, 43, 10.1007/BF02787910 Lieber, 1996, Adenovirus-mediated expression of ribozymes in mice, J Virol, 70, 3153, 10.1128/JVI.70.5.3153-3158.1996 Feng, 1995, Neoplastic reversion accomplished by high efficiency adenoviral-mediated delivery of an anti-ras ribozyme, Cancer Res, 55, 2024 Towbin, 1997, Special topics in gene delivery: adenoviruses and the myocardium, 377