메뉴 건너뛰기




Volumn 17, Issue 11, 2010, Pages 1355-1362

Improvement of the mdx mouse dystrophic phenotype by systemic in utero AAV8 delivery of a minidystrophin gene

Author keywords

Duchenne Gene Therapy; Muscle; Skeletal Dependovirus Fetal Research Muscular Dystrophy

Indexed keywords

DYSTROPHIN; PARVOVIRUS VECTOR; RECOMBINANT DYSTROPHIN; UNCLASSIFIED DRUG;

EID: 78149469593     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2010.84     Document Type: Article
Times cited : (29)

References (46)
  • 1
    • 0025998134 scopus 로고
    • Population frequencies of inherited neuromuscular diseases-a world survey
    • Emery AE. Population frequencies of inherited neuromuscular diseases-a world survey. Neuromuscul Disord 1991; 1: 19-29.
    • (1991) Neuromuscul Disord , vol.1 , pp. 19-29
    • Emery, A.E.1
  • 3
    • 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
  • 5
    • 12744269885 scopus 로고    scopus 로고
    • Adeno-associated virus-mediated microdystrophin expression protects young mdx muscle from contraction-induced injury
    • Liu M, Yue Y, Harper SQ, Grange RW, Chamberlain JS, Duan D. Adeno-associated virus-mediated microdystrophin expression protects young mdx muscle from contraction-induced injury. Mol Ther 2005; 11: 245-256.
    • (2005) Mol Ther , vol.11 , pp. 245-256
    • Liu, M.1    Yue, Y.2    Harper, S.Q.3    Grange, R.W.4    Chamberlain, J.S.5    Duan, D.6
  • 6
    • 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
  • 7
    • 33745479703 scopus 로고    scopus 로고
    • Antisense oligonucleotide-induced exon skipping restores dystro-phin expression in vitro in a canine model of DMD
    • McClorey G, Moulton HM, Iversen PL, Fletcher S, Wilton SD. Antisense oligonucleotide-induced exon skipping restores dystro-phin expression in vitro in a canine model of DMD. Gene Therapy 2006; 13: 1373-1381.
    • (2006) Gene Therapy , vol.13 , pp. 1373-1381
    • McClorey, G.1    Moulton, H.M.2    Iversen, P.L.3    Fletcher, S.4    Wilton, S.D.5
  • 8
    • 10744233235 scopus 로고    scopus 로고
    • Dystrophic phenotype of canine X-linked muscular dystrophy is mitigated by adenovirus-mediated utrophin gene transfer
    • Cerletti M, Negri T, Cozzi F, Colpo R, Andreetta F, Croci D et al. Dystrophic phenotype of canine X-linked muscular dystrophy is mitigated by adenovirus-mediated utrophin gene transfer. Gene Therapy 2003; 10: 750-757.
    • (2003) Gene Therapy , vol.10 , pp. 750-757
    • Cerletti, M.1    Negri, T.2    Cozzi, F.3    Colpo, R.4    Andreetta, F.5    Croci, D.6
  • 9
    • 54249126862 scopus 로고    scopus 로고
    • Gene therapy using adeno-associated virus vectors
    • Daya S, Berns KI. Gene therapy using adeno-associated virus vectors. Clin Microbiol Rev 2008; 21: 583-593.
    • (2008) Clin Microbiol Rev , vol.21 , pp. 583-593
    • Daya, S.1    Berns, K.I.2
  • 10
    • 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
  • 11
    • 19644378907 scopus 로고    scopus 로고
    • Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart
    • Wang Z, Zhu T, Qiao C, Zhou L, Wang B, Zhang J et al. Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart. Nat Biotechnol 2005; 23: 321-328.
    • (2005) Nat Biotechnol , vol.23 , pp. 321-328
    • Wang, Z.1    Zhu, T.2    Qiao, C.3    Zhou, L.4    Wang, B.5    Zhang, J.6
  • 12
    • 36448994441 scopus 로고    scopus 로고
    • A translational approach for limb vascular delivery of the micro-dystrophin gene without high volume or high pressure for treatment of Duchenne muscular dystrophy
    • Rodino-Klapac LR, Janssen PM, Montgomery CL, Coley BD, Chicoine LG, Clark KR et al. A translational approach for limb vascular delivery of the micro-dystrophin gene without high volume or high pressure for treatment of Duchenne muscular dystrophy. J Transl Med 2007; 5: 45.
    • (2007) J Transl Med , vol.5 , pp. 45
    • Rodino-Klapac, L.R.1    Janssen, P.M.2    Montgomery, C.L.3    Coley, B.D.4    Chicoine, L.G.5    Clark, K.R.6
  • 13
    • 0002845186 scopus 로고    scopus 로고
    • Embryonic origins of skeletal muscles
    • Engel AG, Franzini-Armstrong C (eds) McGraw-Hill: NewYork USA
    • Emerson CP, Hauschka SD. Embryonic Origins of Skeletal Muscles. In: Engel AG, Franzini-Armstrong C (eds). Myology. McGraw-Hill: NewYork USA, 2004, pp 3-44.
    • (2004) Myology , pp. 3-44
    • Emerson, C.P.1    Hauschka, S.D.2
  • 14
    • 34547653028 scopus 로고    scopus 로고
    • Cellular heterogeneity during vertebrate skeletal muscle development
    • Biressi S, Molinaro M, Cossu G. Cellular heterogeneity during vertebrate skeletal muscle development. Dev Biol 2007; 308: 281-293.
    • (2007) Dev Biol , vol.308 , pp. 281-293
    • Biressi, S.1    Molinaro, M.2    Cossu, G.3
  • 16
    • 0024447745 scopus 로고
    • Cellular insertion of primary and secondary myotubes in embryonic rat muscles
    • Duxson MJ, Usson Y. Cellular insertion of primary and secondary myotubes in embryonic rat muscles. Development 1989; 107: 243-251.
    • (1989) Development , vol.107 , pp. 243-251
    • Duxson, M.J.1    Usson, Y.2
  • 17
    • 0023909197 scopus 로고
    • Cytoarchitecture of the fetal murine soleus muscle
    • Ontell M, Bourke D, Hughes D. Cytoarchitecture of the fetal murine soleus muscle. Am J Anat 1988; 181: 267-278.
    • (1988) Am J Anat , vol.181 , pp. 267-278
    • Ontell, M.1    Bourke, D.2    Hughes, D.3
  • 18
    • 0021150350 scopus 로고
    • Organogenesis of the mouse extensor digitorum longus muscle: A quantitative study
    • Ontell M, Kozeka K. Organogenesis of the mouse extensor digitorum logus muscle: a quantitative study. Am J Anat 1984; 171: 149-161. (Pubitemid 14023405)
    • (1984) American Journal of Anatomy , vol.171 , Issue.2 , pp. 149-161
    • Ontell, M.1    Kozeka, K.2
  • 19
    • 0021200347 scopus 로고
    • The organogenesis of murine striated muscle: A cytoarchitectural study
    • Ontell M, Kozeka K. The organogenesis of murine striated muscle: a cytoarchitectural study. Am J Anat 1984; 171: 133-148. (Pubitemid 14023404)
    • (1984) American Journal of Anatomy , vol.171 , Issue.2 , pp. 133-148
    • Ontell, M.1    Kozeka, K.2
  • 20
    • 5444227748 scopus 로고    scopus 로고
    • Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy
    • Waddington SN, Nivsarkar MS, Mistry AR, Buckley SM, Kemball-Cook G, Mosley KL et al. Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy. Blood 2004; 104: 2714-2721.
    • (2004) Blood , vol.104 , pp. 2714-2721
    • Waddington, S.N.1    Nivsarkar, M.S.2    Mistry, A.R.3    Buckley, S.M.4    Kemball-Cook, G.5    Mosley, K.L.6
  • 21
    • 0037441596 scopus 로고    scopus 로고
    • In utero gene transfer of human factor IX to fetal mice can induce postnatal tolerance of the exogenous clotting factor
    • Waddington SN, Buckley SM, Nivsarkar M, Jezzard S, Schneider H, Dahse Tet al. In utero gene transfer of human factor IX to fetal mice can induce postnatal tolerance of the exogenous clotting factor. Blood 2003; 101: 1359-1366.
    • (2003) Blood , vol.101 , pp. 1359-1366
    • Waddington, S.N.1    Buckley, S.M.2    Nivsarkar, M.3    Jezzard, S.4    Schneider, H.5    Dahse, T.6
  • 22
    • 34548068776 scopus 로고    scopus 로고
    • Persistent expression of hF IX after tolerance induction by in utero or neonatal administration of AAV-1-F IX in hemophilia B mice
    • Sabatino DE, Mackenzie TC, Peranteau W, Edmonson S, Campagnoli C, Liu YL et al. Persistent expression of hF IX After tolerance induction by in utero or neonatal administration of AAV-1-F IX in hemophilia B mice. Mol Ther 2007; 15: 1677-1685.
    • (2007) Mol Ther , vol.15 , pp. 1677-1685
    • Sabatino, D.E.1    MacKenzie, T.C.2    Peranteau, W.3    Edmonson, S.4    Campagnoli, C.5    Liu, Y.L.6
  • 23
    • 0029797726 scopus 로고    scopus 로고
    • The basal lamina is a physical barrier to herpes simplex virus-mediated gene delivery to mature muscle fibers
    • Huard J, Feero WG, Watkins SC, Hoffman EP, Rosenblatt DJ, Glorioso JC. The basal lamina is a physical barrier to herpes simplex virus-mediated gene delivery to mature muscle fibers. J Virol 1996; 70: 8117-8123.
    • (1996) J Virol , vol.70 , pp. 8117-8123
    • Huard, J.1    Feero, W.G.2    Watkins, S.C.3    Hoffman, E.P.4    Rosenblatt, D.J.5    Glorioso, J.C.6
  • 25
    • 70249117075 scopus 로고    scopus 로고
    • Systemic delivery of AAV8 in utero results in gene expression in diaphragm and limb muscle: Treatment implications for muscle disorders
    • Koppanati BM, Li J, Xiao X, Clemens PR. Systemic delivery of AAV8 in utero results in gene expression in diaphragm and limb muscle: treatment implications for muscle disorders. Gene Therapy 2009; 16: 1130-1137.
    • (2009) Gene Therapy , vol.16 , pp. 1130-1137
    • Koppanati, B.M.1    Li, J.2    Xiao, X.3    Clemens, P.R.4
  • 26
    • 0036087342 scopus 로고    scopus 로고
    • Function and genetics of dystrophin and dystrophin-related proteins in muscle
    • Blake DJ, Weir A, Newey SE, Davies KE. Function and genetics of dystrophin and dystrophin-related proteins in muscle. Physiol Rev 2002; 82: 291-329.
    • (2002) Physiol Rev , vol.82 , pp. 291-329
    • Blake, D.J.1    Weir, A.2    Newey, S.E.3    Davies, K.E.4
  • 27
    • 0035190381 scopus 로고    scopus 로고
    • The dystrophin-glycoprotein complex, cellular signaling, and the regulation of cell survival in the muscular dystrophies
    • Rando TA. The dystrophin-glycoprotein complex, cellular signaling, and the regulation of cell survival in the muscular dystrophies. Muscle Nerve 2001; 24: 1575-1594.
    • (2001) Muscle Nerve , vol.24 , pp. 1575-1594
    • Rando, T.A.1
  • 28
    • 0023091942 scopus 로고
    • The mutant mdx: Inherited myopathy in the mouse Morphological studies of nerves, muscles and end-plates
    • Torres LF, Duchen LW. The mutant mdx: inherited myopathy in the mouse Morphological studies of nerves, muscles and end-plates. Brain 1987; 110(Part 2): 269-299.
    • (1987) Brain , vol.110 , Issue.PART 2 , pp. 269-299
    • Torres, L.F.1    Duchen, L.W.2
  • 29
    • 0031914186 scopus 로고    scopus 로고
    • Aging normal and dystrophic mouse muscle: Analysis of myogenicity in cultures of living single fibers
    • Bockhold KJ, Rosenblatt JD, Partridge TA. Aging normal and dystrophic mouse muscle: analysis of myogenicity in cultures of living single fibers. Muscle Nerve 1998; 21: 173-183.
    • (1998) Muscle Nerve , vol.21 , pp. 173-183
    • Bockhold, K.J.1    Rosenblatt, J.D.2    Partridge, T.A.3
  • 31
    • 0036021778 scopus 로고    scopus 로고
    • Functional characteristics of dystrophic skeletal muscle: Insights from animal models
    • Watchko JF, O'Day TL, Hoffman EP. Functional characteristics of dystrophic skeletal muscle: insights from animal models. J Appl Physiol 2002; 93: 407-417.
    • (2002) J Appl Physiol , vol.93 , pp. 407-417
    • Watchko, J.F.1    O'Day, T.L.2    Hoffman, E.P.3
  • 32
    • 33745121193 scopus 로고    scopus 로고
    • Genetic correction of the fetal brain increases the lifespan of mice with the severe multisystemic disease mucopolysaccharidosis type VII
    • Karolewski BA, Wolfe JH. Genetic correction of the fetal brain increases the lifespan of mice with the severe multisystemic disease mucopolysaccharidosis type VII. Mol Ther 2006; 14: 14-24.
    • (2006) Mol Ther , vol.14 , pp. 14-24
    • Karolewski, B.A.1    Wolfe, J.H.2
  • 33
    • 13644258459 scopus 로고    scopus 로고
    • Widespread gene transduction to the central nervous system by adenovirus in utero: Implication for prenatal gene therapy to brain involvement of lysosomal storage disease
    • Shen JS, Meng XL, Maeda H, Ohashi T, Eto Y. Widespread gene transduction to the central nervous system by adenovirus in utero: implication for prenatal gene therapy to brain involvement of lysosomal storage disease. J Gene Med 2004; 6: 1206-1215.
    • (2004) J Gene Med , vol.6 , pp. 1206-1215
    • Shen, J.S.1    Meng, X.L.2    Maeda, H.3    Ohashi, T.4    Eto, Y.5
  • 34
    • 0142181993 scopus 로고    scopus 로고
    • Long-term correction of bilirubin UDPglucuronyl-transferase deficiency in rats by in utero lentiviral gene transfer
    • Seppen J, van der Rijt R, Looije N, van Til NP, Lamers WH, Oude Elferink RP. Long-term correction of bilirubin UDPglucuronyl-transferase deficiency in rats by in utero lentiviral gene transfer. Mol Ther 2003; 8: 593-599.
    • (2003) Mol Ther , vol.8 , pp. 593-599
    • Seppen, J.1    Van Der Rijt, R.2    Looije, N.3    Van Til, N.P.4    Lamers, W.H.5    Oude Elferink, R.P.6
  • 36
    • 0031040502 scopus 로고    scopus 로고
    • Reversal of cystic fibrosis phenotype in mice by gene therapy in utero
    • Larson JE, Morrow SL, Happel L, Sharp JF, Cohen JC. Reversal of cystic fibrosis phenotype in mice by gene therapy in utero. Lancet 1997; 349: 619-620.
    • (1997) Lancet , vol.349 , pp. 619-620
    • Larson, J.E.1    Morrow, S.L.2    Happel, L.3    Sharp, J.F.4    Cohen, J.C.5
  • 37
    • 3242886999 scopus 로고    scopus 로고
    • Rescue of enzyme deficiency in embryonic diaphragm in a mouse model of metabolic myopathy: Pompe disease
    • Rucker M, Fraites Jr TJ, Porvasnik SL, Lewis MA, Zolotukhin I, Cloutier DA et al. Rescue of enzyme deficiency in embryonic diaphragm in a mouse model of metabolic myopathy: Pompe disease. Development 2004; 131: 3007-3019.
    • (2004) Development , vol.131 , pp. 3007-3019
    • Rucker, M.1    Fraites Jr., T.J.2    Porvasnik, S.L.3    Lewis, M.A.4    Zolotukhin, I.5    Cloutier, D.A.6
  • 38
    • 44349170706 scopus 로고    scopus 로고
    • Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection
    • Zincarelli C, Soltys S, Rengo G, Rabinowitz JE. Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection. Mol Ther 2008; 16: 1073-1080.
    • (2008) Mol Ther , vol.16 , pp. 1073-1080
    • Zincarelli, C.1    Soltys, S.2    Rengo, G.3    Rabinowitz, J.E.4
  • 39
    • 46249131028 scopus 로고    scopus 로고
    • Ability of adeno-associated virus serotype 8-mediated hepatic expression of acid alpha-glucosidase to correct the biochemical and motor function deficits of presymptomatic and symptomatic Pompe mice
    • Ziegler RJ, Bercury SD, Fidler J, Zhao MA, Foley J, Taksir TV et al. Ability of adeno-associated virus serotype 8-mediated hepatic expression of acid alpha-glucosidase to correct the biochemical and motor function deficits of presymptomatic and symptomatic Pompe mice. Hum Gene Ther 2008; 19: 609-621.
    • (2008) Hum Gene Ther , vol.19 , pp. 609-621
    • Ziegler, R.J.1    Bercury, S.D.2    Fidler, J.3    Zhao, M.A.4    Foley, J.5    Taksir, T.V.6
  • 40
    • 10944239670 scopus 로고    scopus 로고
    • Efficacy of an adeno-associated virus 8-pseudotyped vector in glycogen storage disease type II
    • Sun B, Zhang H, Franco LM, Young SP, Schneider A, Bird A et al. Efficacy of an adeno-associated virus 8-pseudotyped vector in glycogen storage disease type II. Mol Ther 2005; 11: 57-65.
    • (2005) Mol Ther , vol.11 , pp. 57-65
    • Sun, B.1    Zhang, H.2    Franco, L.M.3    Young, S.P.4    Schneider, A.5    Bird, A.6
  • 41
    • 41149152962 scopus 로고    scopus 로고
    • Myostatin propeptide gene delivery by adeno-associated virus serotype 8 vectors enhances muscle growth and ameliorates dystrophic phenotypes in mdx mice
    • Qiao C, Li J, Jiang J, Zhu X, Wang B, Li J et al. Myostatin propeptide gene delivery by adeno-associated virus serotype 8 vectors enhances muscle growth and ameliorates dystrophic phenotypes in mdx mice. Hum Gene Ther 2008; 19: 241-254.
    • (2008) Hum Gene Ther , vol.19 , pp. 241-254
    • Qiao, C.1    Li, J.2    Jiang, J.3    Zhu, X.4    Wang, B.5    Li, J.6
  • 42
    • 47549101040 scopus 로고    scopus 로고
    • A canine minidystrophin is functional and therapeutic in mdx mice
    • Wang B, Li J, Qiao C, Chen C, Hu P, Zhu X et al. A canine minidystrophin is functional and therapeutic in mdx mice. Gene Therapy 2008; 15: 1099-1106.
    • (2008) Gene Therapy , vol.15 , pp. 1099-1106
    • Wang, B.1    Li, J.2    Qiao, C.3    Chen, C.4    Hu, P.5    Zhu, X.6
  • 43
    • 2642642141 scopus 로고    scopus 로고
    • Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus
    • Xiao X, Li J, Samulski RJ. Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus. J Virol 1998; 72: 2224-2232.
    • (1998) J Virol , vol.72 , pp. 2224-2232
    • Xiao, X.1    Li, J.2    Samulski, R.J.3
  • 44
    • 0030959261 scopus 로고    scopus 로고
    • Role for highly regulated rep gene expression in adeno-associated virus vector production
    • Li J, Samulski RJ, Xiao X. Role for highly regulated rep gene expression in adeno-associated virus vector production. J Virol 1997; 71: 5236-5243.
    • (1997) J Virol , vol.71 , pp. 5236-5243
    • Li, J.1    Samulski, R.J.2    Xiao, X.3
  • 45
    • 11844269203 scopus 로고    scopus 로고
    • Comparison of high-capacity and first-generation adeno-viral vector gene delivery to murine muscle in utero
    • Bilbao R, Reay DP, Wu E, Zheng H, Biermann V, Kochanek S et al. Comparison of high-capacity and first-generation adeno-viral vector gene delivery to murine muscle in utero. Gene Therapy 2005; 12: 39-47.
    • (2005) Gene Therapy , vol.12 , pp. 39-47
    • Bilbao, R.1    Reay, D.P.2    Wu, E.3    Zheng, H.4    Biermann, V.5    Kochanek, S.6
  • 46
    • 0031826294 scopus 로고    scopus 로고
    • A new blocking method for application of murine monoclonal antibody to mouse tissue sections
    • Lu QL, Partridge TA. A new blocking method for application of murine monoclonal antibody to mouse tissue sections. J Histochem Cytochem 1998; 46: 977-984.
    • (1998) J Histochem Cytochem , vol.46 , pp. 977-984
    • Lu, Q.L.1    Partridge, T.A.2


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.