Gene therapy for Duchenne muscular dystrophy

Bibliography

• Emery AEH. Duchenne muscular dystrophy. 3rd edition. Oxford University Press; Oxford, New York: 2003
   Google Scholar
• Davie AM, Emery AE. Estimation of proportion of new mutants among cases of Duchenne muscular dystrophy. J Med Genet 1978;15:339-45
   PubMed Web of Science ®Google Scholar
• Darras BT, Blattner P, Harper JF, et al. Intragenic deletions in 21 Duchenne muscular dystrophy (DMD)/Becker muscular dystrophy (BMD) families studied with the dystrophin cDNA: location of breakpoints on HindIII and BglII exon-containing fragment maps, meiotic and mitotic origin of the mutations. Am J Hum Genet 1988;43:620-9
   PubMed Web of Science ®Google Scholar
• Darras BT, Jones HR. Diagnosis of pediatric neuromuscular disorders in the era of DNA analysis. Ped Neurol 2000;23:289-300
   Web of Science ®Google Scholar
• Koenig M, Beggs AH, Moyer M, et al. The molecular basis for Duchenne versus Becker muscular dystrophy: correlation of severity with type of deletion. Am J Hum Genet 1989;45:498-506
   PubMed Web of Science ®Google Scholar
• Chamberlain JS, Grant SG, Reeves AA, et al. Regional localization of the murine Duchenne muscular dystrophy gene on the mouse X chromosome. Somat Cell Molec Genet 1987;13:671-8
   PubMedGoogle Scholar
• Chamberlain JS, Chamberlain JR, Fenwick RG, et al. Diagnosis of Duchenne and Becker muscular dystrophies by polymerase chain reaction: A multicenter study. JAMA 1992;267:2609-15
   PubMed Web of Science ®Google Scholar
• Monaco AP, Bertelson CJ, Liechti-Gallati S, et al. An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus. Genomics 1988;2:90-5
   PubMed Web of Science ®Google Scholar
• Baumbach LL, Chamberlain JS, Ward PA, et al. Molecular and clinical correlations of deletions leading to Duchenne and Becker muscular dystrophies. Neurology 1989;39:465-74
   PubMed Web of Science ®Google Scholar
• England SB, Nicholson LV, Johnson MA, et al. Very mild muscular dystrophy associated with the deletion of 46% of dystrophin. Nature 1990;343:180-2
   PubMed Web of Science ®Google Scholar
• Phelps SF, Hauser MA, Cole NM, et al. Expression of full-length and truncated dystrophin mini-genes in transgenic mdx mice. Hum Molec Genet 1995;4:1251-8
   PubMed Web of Science ®Google Scholar
• Hoffman EP, Fischbeck KH, Brown RH, et al. Characterization of dystrophin in muscle-biopsy specimens from patients with Duchenne’s or Becker’s muscular dystrophy. N Engl J Med 1988;318:1363-8
   PubMed Web of Science ®Google Scholar
• Ervasti JM. Structure and function of the dystrophin-glycoprotein complex. In: Winder SJ, editor. Molecular mechanisms of muscular dystrophies. Landes Biosciences; Georgetown: 2006. p. 1-13
   Google Scholar
• Ramaswamy KS, Palmer ML, van der Meulen JH, et al. Lateral transmission of force is impaired in skeletal muscles of dystrophic mice and very old rats. J Physiol 2011;589:1195-208
   PubMed Web of Science ®Google Scholar
• Abmayr S, Chamberlain J. The structure and function of dystrophin. In: Winder SJ, editor. Molecular mechanisms of muscular dystrophies. Landes Biosciences; Georgetown: 2006. p. 14-34
   Google Scholar
• Lai Y, Thomas GD, Yue Y, et al. Dystrophins carrying spectrin-like repeats 16 and 17 anchor nNOS to the sarcolemma and enhance exercise performance in a mouse model of muscular dystrophy. J Clin Invest 2009;119:624-35
   PubMed Web of Science ®Google Scholar
• Winder SJ. The membrane-cytoskeleton interface: the role of dystrophin and utrophin. J Mus Res Cell Motil 1997;18:617-29
   PubMed Web of Science ®Google Scholar
• Lai Y, Zhao J, Yue Y, Duan D. Alpha2 and alpha3 helices of dystrophin R16 and R17 frame a microdomain in the alpha1 helix of dystrophin R17 for neuronal NOS binding. PNAS 2013;110:525-30
   PubMed Web of Science ®Google Scholar
• Prins KW, Humston JL, Mehta A, et al. Dystrophin is a microtubule-associated protein. J Cell Biol 2009;186:363-9
   PubMed Web of Science ®Google Scholar
• Bhosle RC, Michele DE, Campbell KP, et al. Interactions of intermediate filament protein synemin with dystrophin and utrophin. Biochem Biophys Res Commun 2006;346:768-77
   PubMed Web of Science ®Google Scholar
• Stone MR, O’Neill A, Catino D, Bloch RJ. Specific interaction of the actin-binding domain of dystrophin with intermediate filaments containing keratin 19. Mol Biol Cell 2005;16:4280-93
   PubMed Web of Science ®Google Scholar
• Rezniczek GA, Konieczny P, Nikolic B, et al. Plectin 1f scaffolding at the sarcolemma of dystrophic (mdx) muscle fibers through multiple interactions with beta-dystroglycan. J Cell Biol 2007;176:965-77
   PubMed Web of Science ®Google Scholar
• Ayalon G, Davis JQ, Scotland PB, Bennett V. An ankyrin-based mechanism for functional organization of dystrophin and dystroglycan. Cell 2008;135:1189-200
   PubMed Web of Science ®Google Scholar
• Ishikawa-Sakurai M, Yoshida M, Imamura M, et al. ZZ domain is essentially required for the physiological binding of dystrophin and utrophin to beta-dystroglycan. Hum Molec Genet 2004;13:693-702
   PubMed Web of Science ®Google Scholar
• Ozawa E, Nishino I, Nonaka I. Sarcolemmopathy: muscular dystrophies with cell membrane defects. Brain Pathol 2001;11:218-30
   PubMed Web of Science ®Google Scholar
• Ibraghimov-Beskrovnaya O, Ervasti JM, Leveille CJ, et al. Primary structure of dystrophin-associated glycoproteins linking dystrophin to the extracellular matrix. Nature 1992;355:696-702
   PubMed Web of Science ®Google Scholar
• Burkin DJ, Kaufman SJ. The alpha7beta1 integrin in muscle development and disease. Cell Tiss Res 1999;296:183-90
   PubMed Web of Science ®Google Scholar
• Noguchi S, Wakabayashi E, Imamura M, et al. Developmental expression of sarcoglycan gene products in cultured myocytes. Biochem Biophys Res Commun 1999;262:88-93
   PubMed Web of Science ®Google Scholar
• Sakamoto A, Ono K, Abe M, et al. 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. PNAS 1997;94:13873-8
   PubMed Web of Science ®Google Scholar
• Crosbie RH, Heighway J, Venzke DP, et al. Sarcospan, the 25-kDa transmembrane component of the dystrophin-glycoprotein complex. J Biol Chem 1997;272:31221-4
   PubMed Web of Science ®Google Scholar
• Marshall JL, Crosbie-Watson RH. Sarcospan: a small protein with large potential for Duchenne muscular dystrophy. Skel Mus 2013;3:1
   PubMedGoogle Scholar
• Crosbie RH, Dovico SA, Flanagan JD, et al. Characterization of aquaporin-4 in muscle and muscular dystrophy. FASEB J 2002;16:943-9
   PubMed Web of Science ®Google Scholar
• Grady RM, Grange RW, Lau KS, et al. Role for alpha-dystrobrevin in the pathogenesis of dystrophin-dependent muscular dystrophies. Nat Cell Biol 1999;1:215-20
   PubMed Web of Science ®Google Scholar
• Adams ME, Kramarcy N, Krall SP, et al. Absence of alpha-syntrophin leads to structurally aberrant neuromuscular synapses deficient in utrophin. J Cell Biol 2000;150:1385-98
   PubMed Web of Science ®Google Scholar
• Kameya S, Miyagoe Y, Nonaka I, et al. alpha1-syntrophin gene disruption results in the absence of neuronal-type nitric-oxide synthase at the sarcolemma but does not induce muscle degeneration. J Biol Chem 1999;274:2193-200
   PubMed Web of Science ®Google Scholar
• Adams ME, Tesch Y, Percival JM, et al. Differential targeting of nNOS and AQP4 to dystrophin-deficient sarcolemma by membrane-directed alpha-dystrobrevin. J Cell Sci 2008;121:48-54
   PubMed Web of Science ®Google Scholar
• Mendell JR, Campbell K, Rodino-Klapac L, et al. Dystrophin immunity in Duchenne’s muscular dystrophy. New Engl J Med 2010;363:1429-37
   PubMed Web of Science ®Google Scholar
• Mays LE, Wilson JM. The complex and evolving story of T cell activation to AAV vector-encoded transgene products. Mol Ther 2011;19:16-27
   PubMed Web of Science ®Google Scholar
• Atchison RW, Casto BC, Hammon WM. Adenovirus-associated defective virus particles. Science 1965;149:754-6
   PubMed Web of Science ®Google Scholar
• McLaughlin SK, Collis P, Hermonat PL, Muzyczka N. Adeno-associated virus general transduction vectors: analysis of proviral structures. J Virol 1988;62:1963-73
   PubMed Web of Science ®Google Scholar
• Inagaki K, Lewis SM, Wu X, et al. DNA palindromes with a modest arm length of greater, similar 20 base pairs are a significant target for recombinant adeno-associated virus vector integration in the liver, muscles, and heart in mice. J Virol 2007;81:11290-303
   PubMed Web of Science ®Google Scholar
• Nakai H, Montini E, Fuess S, et al. AAV serotype 2 vectors preferentially integrate into active genes in mice. Nature Genet 2003;34:297-302
   PubMed Web of Science ®Google Scholar
• Arnett LHA, Konieczny P, Ramos JN, et al. Adeno-associated viral vectors do not efficiently target muscle satellite cells. Mol Ther - Meth Clin Develop 2014;1:14038
   PubMed Web of Science ®Google Scholar
• Harper SQ, Hauser MA, DelloRusso C, et al. Modular flexibility of dystrophin: Implications for gene therapy of Duchenne muscular dystrophy. Nat Med 2002;8:253-61
   PubMed Web of Science ®Google Scholar
• Yue Y, Li Z, Harper SQ, et al. Microdystrophin gene therapy of cardiomyopathy restores dystrophin-glycoprotein complex and improves sarcolemma integrity in the mdx mouse heart. Circulation 2003;108:1626-32
   PubMed Web of Science ®Google Scholar
• Wang Z, Storb R, Halbert CL, et al. Successful regional delivery and long-term expression of a dystrophin gene in canine muscular dystrophy: a preclinical model for human therapies. Mol Ther 2012;20:1501-7
   PubMed Web of Science ®Google Scholar
• Townsend D, Blankinship MJ, Allen JM, et al. Systemic administration of micro-dystrophin restores cardiac geometry and prevents dobutamine-induced cardiac pump failure. Mol Ther 2007;15:1086-92
   PubMed Web of Science ®Google Scholar
• Gregorevic P, Allen JM, Minami E, et al. rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice. Nat Med 2006;12:787-9
   PubMed Web of Science ®Google Scholar
• Bostick B, Yue Y, Lai Y, et al. Adeno-associated virus serotype-9 microdystrophin gene therapy ameliorates electrocardiographic abnormalities in mdx mice. Hum Gene Ther 2008;19:851-7
   PubMed Web of Science ®Google Scholar
• Shin JH, Pan X, Hakim CH, et al. Microdystrophin ameliorates muscular dystrophy in the canine model of Duchenne muscular dystrophy. Mol Ther 2013;21:750-7
   PubMed Web of Science ®Google Scholar
• Crawford GE, Lu QL, Partridge TA, Chamberlain JS. Suppression of revertant fibers in mdx mice by expression of a functional dystrophin. Hum Mol Genet 2001;10:2745-50
   PubMed Web of Science ®Google Scholar
• Warner LE, DelloRusso C, Crawford RW, et al. Expression of Dp260 in muscle tethers the actin cytoskeleton to the dystrophin-glycoprotein complex and partially prevents dystrophy. Hum Mol Genet 2002;11:1095-105
   PubMed Web of Science ®Google Scholar
• Corrado K, Rafael JA, Mills PL, et al. Transgenic mdx mice expressing dystrophin with a deletion in the actin-binding domain display a "mild Becker" phenotype. J Cell Biol 1996;134:873-84
   PubMed Web of Science ®Google Scholar
• Banks GB, Gregorevic P, Allen JM, et al. Functional capacity of dystrophins carrying deletions in the N-terminal actin-binding domain. Hum Mol Genet 2007;16:2105-13
   PubMed Web of Science ®Google Scholar
• McCabe ER, Towbin J, Chamberlain J, et al. Complementary DNA probes for the Duchenne muscular dystrophy locus demonstrate a previously undetectable deletion in a patient with dystrophic myopathy, glycerol kinase deficiency, and congenital adrenal hypoplasia. J Clin Invest 1989;83:95-9
   PubMed Web of Science ®Google Scholar
• Koenig M, Beggs AH, Moyer M, et al. The molecular basis for Duchenne versus Becker muscular dystrophy: correlation of severity with type of deletion. Amer J Hum Genet 1989;45:498-506
   PubMed Web of Science ®Google Scholar
• Beggs AH, Hoffman EP, Snyder JR, et al. Exploring the molecular basis for variability among patients with Becker muscular dystrophy: dystrophin gene and protein studies. Amer J Hum Genet 1991;49:54-67
   PubMed Web of Science ®Google Scholar
• Harper SQ, Hauser MA, DelloRusso C, et al. Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy. Nature Med 2002;8:253-61
   PubMed Web of Science ®Google Scholar
• Wang B, Li J, Xiao X. Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model. PNAS 2000;97:13714-19
   PubMed Web of Science ®Google Scholar
• Odom GL, Gregorevic P, Allen JM, Chamberlain JS. Gene therapy of mdx mice with large truncated dystrophins generated by recombination using rAAV6. Mol Ther 2011;19:36-45
   PubMed Web of Science ®Google Scholar
• Lostal W, Kodippili K, Yue Y, Duan D. Full-length dystrophin reconstitution with adeno-associated viral vectors. Hum Gene Ther 2014;25(6):552-62
   PubMed Web of Science ®Google Scholar
• Koo T, Popplewell L, Athanasopoulos T, Dickson G. Triple trans-splicing adeno-associated virus vectors capable of transferring the coding sequence for full-length dystrophin protein into dystrophic mice. Hum Gene Ther 2014;25:98-108
   PubMed Web of Science ®Google Scholar
• Gregorevic P, Blankinship MJ, Allen JM, et al. Systemic delivery of genes to striated muscles using adeno-associated viral vectors. Nat Med 2004;10:828-34
   PubMed Web of Science ®Google Scholar
• Le Guiner C, Montus M, Servais L, et al. Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients. Mol Ther 2014;22:1923-35
   PubMed Web of Science ®Google Scholar
• Yue Y, Ghosh A, Long C, et al. A single intravenous injection of adeno-associated virus serotype-9 leads to whole body skeletal muscle transduction in dogs. Mol Ther 2008;16:1944-52
   PubMed Web of Science ®Google Scholar
• Kornegay JN, Li J, Bogan JR, et al. Widespread muscle expression of an AAV9 human mini-dystrophin vector after intravenous injection in neonatal dystrophin-deficient dogs. Mol Ther 2010;18:1501-8
   PubMed Web of Science ®Google Scholar
• Pan X, Yue Y, Zhang K, et al. Long-term robust myocardial transduction of the dog heart from a peripheral vein by adeno-associated virus serotype-8. Hum Gene Ther 2013;24:584-94
   PubMed Web of Science ®Google Scholar
• Salva MZ, Himeda CL, Tai PW, et al. Design of tissue-specific regulatory cassettes for high-level rAAV-mediated expression in skeletal and cardiac muscle. Mol Ther 2007;15:320-9
   PubMed Web of Science ®Google Scholar
• Tai PW, Smith CL, Angello JC, Hauschka SD. Analysis of fiber-type differences in reporter gene expression of beta-gal transgenic muscle. Meth Mol Biol 2012;798:445-59
   PubMedGoogle Scholar
• Swain SL, McKinstry KK, Strutt TM. Expanding roles for CD4(+) T cells in immunity to viruses. Nat Rev Immunol 2012;12:136-48
   PubMed Web of Science ®Google Scholar
• Lund FE, Randall TD. Effector and regulatory B cells: modulators of CD4+ T cell immunity. Nat Rev Immunol 2010;10:236-47
   PubMed Web of Science ®Google Scholar
• Zaiss AK, Cotter MJ, White LR, et al. Complement is an essential component of the immune response to adeno-associated virus vectors. J Virol 2008;82:2727-40
   PubMed Web of Science ®Google Scholar
• Wright JF, Wellman J, High KA. Manufacturing and regulatory strategies for clinical AAV2-hRPE65. Curr Gene Ther 2010;10:341-9
   PubMed Web of Science ®Google Scholar
• Zhu J, Huang X, Yang Y. The TLR9-MyD88 pathway is critical for adaptive immune responses to adeno-associated virus gene therapy vectors in mice. J Clin Invest 2009;119:2388-98
   PubMed Web of Science ®Google Scholar
• Martino AT, Suzuki M, Markusic DM, et al. The genome of self-complementary adeno-associated viral vectors increases Toll-like receptor 9-dependent innate immune responses in the liver. Blood 2011;117:6459-68
   PubMed Web of Science ®Google Scholar
• Siegal FP. The nature of the principal type 1 interferon-producing cells in human blood. Science 1999;284:1835-7
   PubMed Web of Science ®Google Scholar
• Coccia EM, Severa M, Giacomini E, et al. Viral infection and Toll-like receptor agonists induce a differential expression of type I and lambda interferons in human plasmacytoid and monocyte-derived dendritic cells. Eur J Immunol 2004;34:796-805
   PubMed Web of Science ®Google Scholar
• Salio M, Palmowski MJ, Atzberger A, et al. CpG-matured murine plasmacytoid dendritic cells are capable of in vivo priming of functional CD8 T cell responses to endogenous but not exogenous antigens. J Exp Med 2004;199:567-79
   PubMed Web of Science ®Google Scholar
• Zhang Y, Chirmule N, Gao GP, Wilson J. CD40 Ligand-dependent activation of cytotoxic T lymphocytes by adeno-associated virus vectors in vivo: role of immature dendritic cells. J Virol 2000;74:8003-10
   PubMed Web of Science ®Google Scholar
• Vandenberghe LH, Wang L, Somanathan S, et al. Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid. Nat Med 2006;12:967-71
   PubMed Web of Science ®Google Scholar
• Pien GC, Basner-Tschakarjan E, Hui DJ, et al. Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors. J Clin Inv 2009;119:1688-95
   PubMed Web of Science ®Google Scholar
• Li H, Tuyishime S, Wu TL, et al. Adeno-associated virus vectors serotype 2 induce prolonged proliferation of capsid-specific CD8+ T cells in mice. Mol Ther 2011;19:536-46
   PubMed Web of Science ®Google Scholar
• Li H, Lasaro MO, Jia B, et al. Capsid-specific T-cell responses to natural infections with adeno-associated viruses in humans differ from those of nonhuman primates. Mol Ther 2011;19:2021-30
   PubMed Web of Science ®Google Scholar
• Wang Z, Tapscott SJ, Chamberlain JS, Storb R. Immunity and AAV-mediated gene therapy for muscular dystrophies in large animal models and human trials. Front Microbiol 2011;2:201
   PubMedGoogle Scholar
• Mendell JR, Rodino-Klapac LR, Rosales XQ, et al. Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D. Annals Neurol 2010;68:629-38
   PubMed Web of Science ®Google Scholar
• Rogers GL, Martino AT, Aslanidi GV, et al. Innate immune responses to AAV vectors. Front Microbiol 2011;2:194
   PubMed Web of Science ®Google Scholar
• Faust SM, Bell P, Cutler BJ, et al. CpG-depleted adeno-associated virus vectors evade immune detection. J Clin Inv 2013;123:2994-3001
   PubMed Web of Science ®Google Scholar
• Mays LE, Vandenberghe LH, Xiao R, et al. Adeno-associated virus capsid structure drives CD4-dependent CD8+ T cell response to vector encoded proteins. J Immunol 2009;182:6051-60
   PubMed Web of Science ®Google Scholar
• Le Bon A, Etchart N, Rossmann C, et al. Cross-priming of CD8+ T cells stimulated by virus-induced type I interferon. Nat Immunol 2003;4:1009-15
   PubMed Web of Science ®Google Scholar
• Cousens LP, Peterson R, Hsu S, et al. Two roads diverged: interferon alpha/beta- and interleukin 12-mediated pathways in promoting T cell interferon gamma responses during viral infection. J Exp Med 1999;189:1315-28
   PubMed Web of Science ®Google Scholar
• Mosmann TR, Cherwinski H, Bond MW, et al. Two types of murine helper T cell clone. I. Definition according to profiles of lymphokine activities and secreted proteins. J Immunol 1986;136:2348-57
   PubMed Web of Science ®Google Scholar
• Dorner T, Radbruch A. Antibodies and B cell memory in viral immunity. Immunity 2007;27:384-92
   PubMed Web of Science ®Google Scholar
• Coutelier JP, van der Logt JT, Heessen FW, et al. IgG2a restriction of murine antibodies elicited by viral infections. J Exp Med 1987;165:64-9
   PubMed Web of Science ®Google Scholar
• Xiao W, Chirmule N, Schnell MA, et al. Route of administration determines induction of T-cell-independent humoral responses to adeno-associated virus vectors. Mol Ther 2000;1:323-9
   PubMed Web of Science ®Google Scholar
• Chirmule N, Xiao W, Truneh A, et al. Humoral immunity to adeno-associated virus type 2 vectors following administration to murine and nonhuman primate muscle. J Virol 2000;74:2420-5
   PubMed Web of Science ®Google Scholar
• Sudres M, Cire S, Vasseur V, et al. MyD88 signaling in B cells regulates the production of Th1-dependent antibodies to AAV. Mol Ther 2012;20:1571-81
   PubMed Web of Science ®Google Scholar
• Murphy SL, Li H, Mingozzi F, et al. Diverse IgG subclass responses to adeno-associated virus infection and vector administration. J Med Virol 2009;81:65-74
   PubMed Web of Science ®Google Scholar
• Mingozzi F, Meulenberg JJ, Hui DJ, et al. AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells. Blood 2009;114:2077-86
   PubMed Web of Science ®Google Scholar
• Huard J, Roy R, Bouchard JP, et al. Human myoblast transplantation between immunohistocompatible donors and recipients produces immune reactions. Transplant Proc 1992;24:3049-51
   PubMed Web of Science ®Google Scholar
• Ohtsuka Y, Udaka K, Yamashiro Y, et al. Dystrophin acts as a transplantation rejection antigen in dystrophin-deficient mice: implication for gene therapy. J Immunol 1998;160:4635-40
   PubMed Web of Science ®Google Scholar
• Gilchrist SC, Ontell MP, Kochanek S, Clemens PR. Immune response to full-length dystrophin delivered to Dmd muscle by a high-capacity adenoviral vector. Mol Ther 2002;6:359-68
   PubMed Web of Science ®Google Scholar
• Chicoine LG, Montgomery CL, Bremer WG, et al. Plasmapheresis eliminates the negative impact of AAV antibodies on microdystrophin gene expression following vascular delivery. Mol Ther 2014;22:338-47
   PubMed Web of Science ®Google Scholar
• Nathwani AC, Reiss UM, Tuddenham EG, et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med 2014;371:1994-2004
   PubMed Web of Science ®Google Scholar
• Wang Z, Kuhr CS, Allen JM, et al. Sustained AAV-mediated dystrophin expression in a canine model of Duchenne muscular dystrophy with a brief course of immunosuppression. Mol Ther 2007;15:1160-6
   PubMed Web of Science ®Google Scholar
• Lorain S, Gross DA, Goyenvalle A, et al. Transient immunomodulation allows repeated injections of AAV1 and correction of muscular dystrophy in multiple muscles. Mol Ther 2008;16:541-7
   PubMed Web of Science ®Google Scholar
• Manning WC, Zhou S, Bland MP, et al. Transient immunosuppression allows transgene expression following readministration of adeno-associated viral vectors. Hum Gene Ther 1998;9:477-85
   PubMed Web of Science ®Google Scholar
• Halbert CL, Standaert TA, Wilson CB, Miller AD. Successful readministration of adeno-associated virus vectors to the mouse lung requires transient immunosuppression during the initial exposure. J Virol 1998;72:9795-805
   PubMed Web of Science ®Google Scholar
• McIntosh JH, Cochrane M, Cobbold S, et al. Successful attenuation of humoral immunity to viral capsid and transgenic protein following AAV-mediated gene transfer with a non-depleting CD4 antibody and cyclosporine. Gene Ther 2012;19:78-85
   PubMed Web of Science ®Google Scholar
• Roep BO, Solvason N, Gottlieb PA, et al. Plasmid-encoded proinsulin preserves C-peptide while specifically reducing proinsulin-specific CD8+ T cells in type 1 diabetes. Sci Transl Med 2013;5:191ra82
   PubMed Web of Science ®Google Scholar
• Odom GL, Gregorevic P, Allen JM, et al. Microutrophin delivery through rAAV6 increases lifespan and improves muscle function in dystrophic dystrophin/utrophin-deficient mice. Mol Ther 2008;16:1539-45
   PubMed Web of Science ®Google Scholar