메뉴 건너뛰기




Volumn 6, Issue 3, 2010, Pages 111-116

Gene therapy for muscular dystrophies: Progress and challenges

Author keywords

Duchenne; Gene therapy; Muscular dystrophies

Indexed keywords


EID: 78651507899     PISSN: 17386586     EISSN: 20055013     Source Type: Journal    
DOI: 10.3988/jcn.2010.6.3.111     Document Type: Review
Times cited : (4)

References (46)
  • 1
    • 0025887093 scopus 로고
    • Human dystrophin expression in mdx mice after intramuscular injection of DNA constructs
    • Acsadi G, Dickson G, Love DR, Jani A, Walsh FS, Gurusinghe A, et al. Human dystrophin expression in mdx mice after intramuscular injection of DNA constructs. Nature 1991;352:815-818.
    • (1991) Nature , vol.352 , pp. 815-818
    • Acsadi, G.1    Dickson, G.2    Love, D.R.3    Jani, A.4    Walsh, F.S.5    Gurusinghe, A.6
  • 2
    • 0031753867 scopus 로고    scopus 로고
    • Gene transfer into muscle by electroporation in vivo
    • Aihara H, Miyazaki J. Gene transfer into muscle by electroporation in vivo. Nat Biotechnol 1998;16:867-870.
    • (1998) Nat Biotechnol , vol.16 , pp. 867-870
    • Aihara, H.1    Miyazaki, J.2
  • 4
    • 4644275773 scopus 로고    scopus 로고
    • In vivo gene electrotransfer into skeletal muscle: Effects of plasmid DNA on the occurrence and extent of muscle damage
    • Durieux AC, Bonnefoy R, Busso T, Freyssenet D. In vivo gene electrotransfer into skeletal muscle: effects of plasmid DNA on the occurrence and extent of muscle damage. J Gene Med 2004;6:809-816.
    • (2004) J Gene Med , vol.6 , pp. 809-816
    • Durieux, A.C.1    Bonnefoy, R.2    Busso, T.3    Freyssenet, D.4
  • 5
    • 4444279151 scopus 로고    scopus 로고
    • Factors influencing the efficacy, longevity, and safety of electroporation-assisted plasmid-based gene transfer into mouse muscles
    • Molnar MJ, Gilbert R, Lu Y, Liu AB, Guo A, Larochelle N, et al. Factors influencing the efficacy, longevity, and safety of electroporation-assisted plasmid-based gene transfer into mouse muscles. Mol Ther 2004;10:447-455.
    • (2004) Mol Ther , vol.10 , pp. 447-455
    • Molnar, M.J.1    Gilbert, R.2    Lu, Y.3    Liu, A.B.4    Guo, A.5    Larochelle, N.6
  • 7
    • 0037309533 scopus 로고    scopus 로고
    • Full-length dystrophin cDNA transfer into skeletal muscle of adult mdx mice by electroporation
    • Murakami T, Nishi T, Kimura E, Goto T, Maeda Y, Ushio Y, et al. Full-length dystrophin cDNA transfer into skeletal muscle of adult mdx mice by electroporation. Muscle Nerve 2003;27:237-241.
    • (2003) Muscle Nerve , vol.27 , pp. 237-241
    • Murakami, T.1    Nishi, T.2    Kimura, E.3    Goto, T.4    Maeda, Y.5    Ushio, Y.6
  • 8
    • 0034893580 scopus 로고    scopus 로고
    • Optimisation of electrotransfer of plasmid into skeletal muscle by pretreatment with hyaluronidase--increased expression with reduced muscle damage
    • McMahon JM, Signori E, Wells KE, Fazio VM, Wells DJ. Optimisation of electrotransfer of plasmid into skeletal muscle by pretreatment with hyaluronidase--increased expression with reduced muscle damage. Gene Ther 2001;8:1264-1270.
    • (2001) Gene Ther , vol.8 , pp. 1264-1270
    • McMahon, J.M.1    Signori, E.2    Wells, K.E.3    Fazio, V.M.4    Wells, D.J.5
  • 10
    • 0031935395 scopus 로고    scopus 로고
    • The efficient expression of intravascularly delivered DNA in rat muscle
    • Budker V, Zhang G, Danko I, Williams P, Wolff J. The efficient expression of intravascularly delivered DNA in rat muscle. Gene Ther 1998; 5:272-276.
    • (1998) Gene Ther , vol.5 , pp. 272-276
    • Budker, V.1    Zhang, G.2    Danko, I.3    Williams, P.4    Wolff, J.5
  • 11
    • 4143091589 scopus 로고    scopus 로고
    • Intraarterial delivery of naked plasmid DNA expressing full-length mouse dystrophin in the mdx mouse model of duchenne muscular dystrophy
    • Zhang G, Ludtke JJ, Thioudellet C, Kleinpeter P, Antoniou M, Herweijer H, et al. Intraarterial delivery of naked plasmid DNA expressing full-length mouse dystrophin in the mdx mouse model of duchenne muscular dystrophy. Hum Gene Ther 2004;15:770-782.
    • (2004) Hum Gene Ther , vol.15 , pp. 770-782
    • Zhang, G.1    Ludtke, J.J.2    Thioudellet, C.3    Kleinpeter, P.4    Antoniou, M.5    Herweijer, H.6
  • 12
    • 4344715026 scopus 로고    scopus 로고
    • A facile nonviral method for delivering genes and siRNAs to skeletal muscle of mammalian limbs
    • Hagstrom JE, Hegge J, Zhang G, Noble M, Budker V, Lewis DL, et al. A facile nonviral method for delivering genes and siRNAs to skeletal muscle of mammalian limbs. Mol Ther 2004;10:386-398.
    • (2004) Mol Ther , vol.10 , pp. 386-398
    • Hagstrom, J.E.1    Hegge, J.2    Zhang, G.3    Noble, M.4    Budker, V.5    Lewis, D.L.6
  • 13
    • 6344263893 scopus 로고    scopus 로고
    • Phase I study of dystrophin plasmid-based gene therapy in Duchenne/Becker muscular dystrophy
    • Romero NB, Braun S, Benveniste O, Leturcq F, Hogrel JY, Morris GE, et al. Phase I study of dystrophin plasmid-based gene therapy in Duchenne/Becker muscular dystrophy. Hum Gene Ther 2004;15: 1065-1076.
    • (2004) Hum Gene Ther , vol.15 , pp. 1065-1076
    • Romero, N.B.1    Braun, S.2    Benveniste, O.3    Leturcq, F.4    Hogrel, J.Y.5    Morris, G.E.6
  • 15
    • 0033543994 scopus 로고    scopus 로고
    • Gene transfer into the liver of nonhuman primates with E1-deleted recombinant adenoviral vectors: Safety of readministration
    • Nunes FA, Furth EE, Wilson JM, Raper SE. Gene transfer into the liver of nonhuman primates with E1-deleted recombinant adenoviral vectors: safety of readministration. Hum Gene Ther 1999;10:2515-2526.
    • (1999) Hum Gene Ther , vol.10 , pp. 2515-2526
    • Nunes, F.A.1    Furth, E.E.2    Wilson, J.M.3    Raper, S.E.4
  • 16
    • 0031969428 scopus 로고    scopus 로고
    • Transduction of dendritic cells by DNA viral vectors directs the immune response to transgene products in muscle fibers
    • Jooss K, Yang Y, Fisher KJ, Wilson JM. Transduction of dendritic cells by DNA viral vectors directs the immune response to transgene products in muscle fibers. J Virol 1998;72:4212-4223.
    • (1998) J Virol , vol.72 , pp. 4212-4223
    • Jooss, K.1    Yang, Y.2    Fisher, K.J.3    Wilson, J.M.4
  • 17
    • 0034610364 scopus 로고    scopus 로고
    • Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model
    • Wang B, Li J, Xiao X. Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model. Proc Natl Acad Sci U S A 2000;97:13714-13719.
    • (2000) Proc Natl Acad Sci U S A , vol.97 , pp. 13714-13719
    • Wang, B.1    Li, J.2    Xiao, X.3
  • 18
    • 7044253593 scopus 로고    scopus 로고
    • AAV vector-mediated microdystrophin expression in a relatively small percentage of mdx myofibers improved the mdx phenotype
    • Yoshimura M, Sakamoto M, Ikemoto M, Mochizuki Y, Yuasa K, Miyagoe-Suzuki Y, et al. AAV vector-mediated microdystrophin expression in a relatively small percentage of mdx myofibers improved the mdx phenotype. Mol Ther 2004;10:821-828.
    • (2004) Mol Ther , vol.10 , pp. 821-828
    • Yoshimura, M.1    Sakamoto, M.2    Ikemoto, M.3    Mochizuki, Y.4    Yuasa, K.5    Miyagoe-Suzuki, Y.6
  • 20
    • 50549085053 scopus 로고    scopus 로고
    • Lack of toxicity of alpha-sarcoglycan overexpression supports clinical gene transfer trial in LGMD2D
    • Rodino-Klapac LR, Lee JS, Mulligan RC, Clark KR, Mendell JR. Lack of toxicity of alpha-sarcoglycan overexpression supports clinical gene transfer trial in LGMD2D. Neurology 2008;71:240-247.
    • (2008) Neurology , vol.71 , pp. 240-247
    • Rodino-Klapac, L.R.1    Lee, J.S.2    Mulligan, R.C.3    Clark, K.R.4    Mendell, J.R.5
  • 21
    • 0036892308 scopus 로고    scopus 로고
    • Adeno-associated virus vector-mediated gene transfer into dystrophin-deficient skeletal muscles evokes enhanced immune response against the transgene product
    • Yuasa K, Sakamoto M, Miyagoe-Suzuki Y, Tanouchi A, Yamamoto H, Li J, et al. Adeno-associated virus vector-mediated gene transfer into dystrophin-deficient skeletal muscles evokes enhanced immune response against the transgene product. Gene Ther 2002;9:1576-1588.
    • (2002) Gene Ther , vol.9 , pp. 1576-1588
    • Yuasa, K.1    Sakamoto, M.2    Miyagoe-Suzuki, Y.3    Tanouchi, A.4    Yamamoto, H.5    Li, J.6
  • 22
    • 33747584384 scopus 로고    scopus 로고
    • Long-term expression and repeated administration of AAV type 1, 2 and 5 vectors in skeletal muscle of immunocompetent adult mice
    • Rivière C, Danos O, Douar AM. Long-term expression and repeated administration of AAV type 1, 2 and 5 vectors in skeletal muscle of immunocompetent adult mice. Gene Ther 2006;13:1300-1308.
    • (2006) Gene Ther , vol.13 , pp. 1300-1308
    • Rivière, C.1    Danos, O.2    Douar, A.M.3
  • 23
    • 33745894322 scopus 로고    scopus 로고
    • RAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice
    • Gregorevic P, Allen JM, Minami E, Blankinship MJ, Haraguchi M, Meuse L, et al. rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice. Nat Med 2006;12:787-789.
    • (2006) Nat Med , vol.12 , pp. 787-789
    • Gregorevic, P.1    Allen, J.M.2    Minami, E.3    Blankinship, M.J.4    Haraguchi, M.5    Meuse, L.6
  • 24
    • 33745143956 scopus 로고    scopus 로고
    • Robust systemic transduction with AAV9 vectors in mice: Efficient global cardiac gene transfer superior to that of AAV8
    • Inagaki K, Fuess S, Storm TA, Gibson GA, Mctiernan CF, Kay MA, et al. Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8. Mol Ther 2006;14:45-53.
    • (2006) Mol Ther , vol.14 , pp. 45-53
    • Inagaki, K.1    Fuess, S.2    Storm, T.A.3    Gibson, G.A.4    McTiernan, C.F.5    Kay, M.A.6
  • 25
  • 26
    • 0032924996 scopus 로고    scopus 로고
    • rAAV vector-mediated sarcogylcan gene transfer in a hamster model for limb girdle muscular dystrophy
    • Li J, Dressman D, Tsao YP, Sakamoto A, Hoffman EP, Xiao X. rAAV vector-mediated sarcogylcan gene transfer in a hamster model for limb girdle muscular dystrophy. Gene Ther 1999;6:74-82.
    • (1999) Gene Ther , vol.6 , pp. 74-82
    • Li, J.1    Dressman, D.2    Tsao, Y.P.3    Sakamoto, A.4    Hoffman, E.P.5    Xiao, X.6
  • 27
    • 0023718118 scopus 로고
    • An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus
    • Monaco AP, Bertelson CJ, Liechti-Gallati S, Moser H, Kunkel LM. An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus. Genomics 1988;2:90-95.
    • (1988) Genomics , vol.2 , pp. 90-95
    • Monaco, A.P.1    Bertelson, C.J.2    Liechti-Gallati, S.3    Moser, H.4    Kunkel, L.M.5
  • 28
    • 0031800293 scopus 로고    scopus 로고
    • Modification of splicing in the dystrophin gene in cultured Mdx muscle cells by antisense oligoribonucleotides
    • Dunckley MG, Manoharan M, Villiet P, Eperon IC, Dickson G. Modification of splicing in the dystrophin gene in cultured Mdx muscle cells by antisense oligoribonucleotides. Hum Mol Genet 1998;7: 1083-1090.
    • (1998) Hum Mol Genet , vol.7 , pp. 1083-1090
    • Dunckley, M.G.1    Manoharan, M.2    Villiet, P.3    Eperon, I.C.4    Dickson, G.5
  • 30
    • 32244443828 scopus 로고    scopus 로고
    • Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology
    • Alter J, Lou F, Rabinowitz A, Yin H, Rosenfeld J, Wilton SD, et al. Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology. Nat Med 2006;12:175-177.
    • (2006) Nat Med , vol.12 , pp. 175-177
    • Alter, J.1    Lou, F.2    Rabinowitz, A.3    Yin, H.4    Rosenfeld, J.5    Wilton, S.D.6
  • 31
    • 0042536463 scopus 로고    scopus 로고
    • Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse
    • Lu QL, Mann CJ, Lou F, Bou-Gharios G, Morris GE, Xue SA, et al. Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse. Nat Med 2003;9:1009-1014.
    • (2003) Nat Med , vol.9 , pp. 1009-1014
    • Lu, Q.L.1    Mann, C.J.2    Lou, F.3    Bou-Gharios, G.4    Morris, G.E.5    Xue, S.A.6
  • 32
    • 0141760549 scopus 로고    scopus 로고
    • Enhanced in vivo delivery of antisense oligonucleotides to restore dystrophin expression in adult mdx mouse muscle
    • Wells KE, Fletcher S, Mann CJ, Wilton SD, Wells DJ. Enhanced in vivo delivery of antisense oligonucleotides to restore dystrophin expression in adult mdx mouse muscle. FEBS Lett 2003;552:145-149.
    • (2003) FEBS Lett , vol.552 , pp. 145-149
    • Wells, K.E.1    Fletcher, S.2    Mann, C.J.3    Wilton, S.D.4    Wells, D.J.5
  • 33
    • 4344693568 scopus 로고    scopus 로고
    • Targeted exon skipping in transgenic hDMD mice: A model for direct preclinical screening of human-specific antisense oligonucleotides
    • Bremmer-Bout M, Aartsma-Rus A, de Meijer EJ, Kaman WE, Janson AA, Vossen RH, et al. Targeted exon skipping in transgenic hDMD mice: a model for direct preclinical screening of human-specific antisense oligonucleotides. Mol Ther 2004;10:232-240.
    • (2004) Mol Ther , vol.10 , pp. 232-240
    • Bremmer-Bout, M.1    Aartsma-Rus, A.2    de Meijer, E.J.3    Kaman, W.E.4    Janson, A.A.5    Vossen, R.H.6
  • 34
    • 33646671717 scopus 로고    scopus 로고
    • Intravenous infusion of an antisense oligonucleotide results in exon skipping in muscle dystrophin mRNA of Duchenne muscular dystrophy
    • Takeshima Y, Yagi M, Wada H, Ishibashi K, Nishiyama A, Kakumoto M, et al. Intravenous infusion of an antisense oligonucleotide results in exon skipping in muscle dystrophin mRNA of Duchenne muscular dystrophy. Pediatr Res 2006;59:690-694.
    • (2006) Pediatr Res , vol.59 , pp. 690-694
    • Takeshima, Y.1    Yagi, M.2    Wada, H.3    Ishibashi, K.4    Nishiyama, A.5    Kakumoto, M.6
  • 38
    • 0043092426 scopus 로고    scopus 로고
    • Gentamicin administration in Duchenne patients with premature stop codon. Preliminary results
    • Politano L, Nigro G, Nigro V, Piluso G, Papparella S, Paciello O, et al. Gentamicin administration in Duchenne patients with premature stop codon. Preliminary results. Acta Myol 2003;22:15-21.
    • (2003) Acta Myol , vol.22 , pp. 15-21
    • Politano, L.1    Nigro, G.2    Nigro, V.3    Piluso, G.4    Papparella, S.5    Paciello, O.6
  • 39
    • 0033854425 scopus 로고    scopus 로고
    • Sequence specificity of aminoglycoside-induced stop condon readthrough: Potential implications for treatment of Duchenne muscular dystrophy
    • Howard MT, Shirts BH, Petros LM, Flanigan KM, Gesteland RF, Atkins JF. Sequence specificity of aminoglycoside-induced stop condon readthrough: potential implications for treatment of Duchenne muscular dystrophy. Ann Neurol 2000;48:164-169.
    • (2000) Ann Neurol , vol.48 , pp. 164-169
    • Howard, M.T.1    Shirts, B.H.2    Petros, L.M.3    Flanigan, K.M.4    Gesteland, R.F.5    Atkins, J.F.6
  • 41
    • 33947529670 scopus 로고    scopus 로고
    • Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers
    • Hirawat S, Welch EM, Elfring GL, Northcutt VJ, Paushkin S, Hwang S, et al. Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers. J Clin Pharmacol 2007;47:430-444.
    • (2007) J Clin Pharmacol , vol.47 , pp. 430-444
    • Hirawat, S.1    Welch, E.M.2    Elfring, G.L.3    Northcutt, V.J.4    Paushkin, S.5    Hwang, S.6
  • 42
    • 33847789357 scopus 로고    scopus 로고
    • Dystrophin expression in host muscle following transplantation of muscle precursor cells modified with the phiC31 integrase
    • Quenneville SP, Chapdelaine P, Rousseau J, Tremblay JP. Dystrophin expression in host muscle following transplantation of muscle precursor cells modified with the phiC31 integrase. Gene Ther 2007;14: 514-522.
    • (2007) Gene Ther , vol.14 , pp. 514-522
    • Quenneville, S.P.1    Chapdelaine, P.2    Rousseau, J.3    Tremblay, J.P.4
  • 43
    • 36349034522 scopus 로고    scopus 로고
    • Autologous transplantation of SM/C-2.6(+) satellite cells transduced with micro-dystrophin CS1 cDNA by lentiviral vector into mdx mice
    • Ikemoto M, Fukada S, Uezumi A, Masuda S, Miyoshi H, Yamamoto H, et al. Autologous transplantation of SM/C-2.6(+) satellite cells transduced with micro-dystrophin CS1 cDNA by lentiviral vector into mdx mice. Mol Ther 2007;15:2178-2185.
    • (2007) Mol Ther , vol.15 , pp. 2178-2185
    • Ikemoto, M.1    Fukada, S.2    Uezumi, A.3    Masuda, S.4    Miyoshi, H.5    Yamamoto, H.6
  • 44
    • 36749032678 scopus 로고    scopus 로고
    • Restoration of human dystrophin following transplantation of exon-skipping-engineered DMD patient stem cells into dystrophic mice
    • Benchaouir R, Meregalli M, Farini A, D'Antona G, Belicchi M, Goyenvalle A, et al. Restoration of human dystrophin following transplantation of exon-skipping-engineered DMD patient stem cells into dystrophic mice. Cell Stem Cell 2007;1:646-657.
    • (2007) Cell Stem Cell , vol.1 , pp. 646-657
    • Benchaouir, R.1    Meregalli, M.2    Farini, A.3    D'Antona, G.4    Belicchi, M.5    Goyenvalle, A.6


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.