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Volumn 3, Issue 11, 1996, Pages 965-972

In vivo muscle gene transfer of full-length dystrophin with an adenoviral vector that lacks all viral genes

Author keywords

Adenoviral vector; Duchenne muscular dystrophy; Dystrophin; Gene transfer

Indexed keywords

COMPLEMENTARY DNA; CREATINE KINASE; DYSTROPHIN; RECOMBINANT PROTEIN; UTROPHIN;

EID: 0029857641     PISSN: 09697128     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Article
Times cited : (189)

References (30)
  • 1
    • 0023614188 scopus 로고
    • Dystrophin: The protein product of the Duchenne muscular dystrophy locus
    • Hoffman EP, Brown RH Jr, Kunkel LM. Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell 1987; 51: 919-928.
    • (1987) Cell , vol.51 , pp. 919-928
    • Hoffman, E.P.1    Brown Jr., R.H.2    Kunkel, L.M.3
  • 2
    • 0023925292 scopus 로고
    • The Duchenne muscular dystrophy gene product is localized in sarcolemma of human skeletal muscle
    • Zubrzycka-Gaarn EE et al The Duchenne muscular dystrophy gene product is localized in sarcolemma of human skeletal muscle. Nature 1988; 333: 466-469.
    • (1988) Nature , vol.333 , pp. 466-469
    • Zubrzycka-Gaarn, E.E.1
  • 3
    • 0024432114 scopus 로고
    • Dystrophin diagnosis: Comparison of dystrophin abnormalities by immunofluorescence and immunoblot analyses
    • Arahata K et al. Dystrophin diagnosis: comparison of dystrophin abnormalities by immunofluorescence and immunoblot analyses. Proc Natl Acad Sci USA 1989; 86: 7154-7158.
    • (1989) Proc Natl Acad Sci USA , vol.86 , pp. 7154-7158
    • Arahata, K.1
  • 4
    • 0000801692 scopus 로고
    • Dystrophinopathies
    • Engel AG, Franzini-Armstrong C (eds) McGraw-Hill: New York
    • Engel AG, Yamamoto M, Fischbeck KH. Dystrophinopathies. In: Engel AG, Franzini-Armstrong C (eds). Myology. McGraw-Hill: New York, 1994, pp 1133-1187.
    • (1994) Myology , pp. 1133-1187
    • Engel, A.G.1    Yamamoto, M.2    Fischbeck, K.H.3
  • 5
    • 0023764622 scopus 로고
    • Development of adenovirus vectors for the expression of heterologous genes
    • Berkner KL. Development of adenovirus vectors for the expression of heterologous genes. Biotechniques 1988; 6: 616-629.
    • (1988) Biotechniques , vol.6 , pp. 616-629
    • Berkner, K.L.1
  • 6
    • 0028559768 scopus 로고
    • An efficient and flexible system for construction of adenovirus vectors with insertions or deletions in early regions 1 and 3
    • Bett AJ, Haddara W, Prevec L, Graham FL. An efficient and flexible system for construction of adenovirus vectors with insertions or deletions in early regions 1 and 3. Proc Natl Acad Sci USA 1994; 91: 8802-8806.
    • (1994) Proc Natl Acad Sci USA , vol.91 , pp. 8802-8806
    • Bett, A.J.1    Haddara, W.2    Prevec, L.3    Graham, F.L.4
  • 7
    • 0025159208 scopus 로고
    • Very mild muscular dystrophy associated with the deletion of 46% of dystrophin
    • England SB et al. Very mild muscular dystrophy associated with the deletion of 46% of dystrophin. Nature 1990; 343: 180-182.
    • (1990) Nature , vol.343 , pp. 180-182
    • England, S.B.1
  • 8
    • 0028791744 scopus 로고
    • Recombinant truncated dystrophin minigenes: Construction, expression, and adenoviral delivery
    • Clemens PR et al. Recombinant truncated dystrophin minigenes: construction, expression, and adenoviral delivery. Hum Gene Ther 1995; 6: 1477-1485.
    • (1995) Hum Gene Ther , vol.6 , pp. 1477-1485
    • Clemens, P.R.1
  • 9
    • 0029122523 scopus 로고
    • Expression of full-length and truncated dystrophin mini-genes in transgenic mdx mice
    • Phelps SF et al. Expression of full-length and truncated dystrophin mini-genes in transgenic mdx mice. Hum Mol Genet 1995; 4: 1251-1258.
    • (1995) Hum Mol Genet , vol.4 , pp. 1251-1258
    • Phelps, S.F.1
  • 10
    • 0029122522 scopus 로고
    • Expression of human full-length and minidystrophin in transgenic mdx mice: Implications for gene therapy of Duchenne muscular dystrophy
    • Wells DJ et al Expression of human full-length and minidystrophin in transgenic mdx mice: implications for gene therapy of Duchenne muscular dystrophy. Hum Mol Genet 1995; 4: 1245-1250.
    • (1995) Hum Mol Genet , vol.4 , pp. 1245-1250
    • Wells, D.J.1
  • 11
    • 0027408847 scopus 로고
    • Efficient adenovirus-mediated transfer of a human minidystrophin gene to skeletal muscle of mdx mice
    • Ragot T et al Efficient adenovirus-mediated transfer of a human minidystrophin gene to skeletal muscle of mdx mice. Nature 1993; 361: 647-650.
    • (1993) Nature , vol.361 , pp. 647-650
    • Ragot, T.1
  • 12
    • 0027362435 scopus 로고
    • Long-term correction of mouse dystrophic degeneration by adenovirus-mediated transfer of a minidystrophin gene
    • Vincent N et al. Long-term correction of mouse dystrophic degeneration by adenovirus-mediated transfer of a minidystrophin gene. Nat Genet 1993; 5: 130-134.
    • (1993) Nat Genet , vol.5 , pp. 130-134
    • Vincent, N.1
  • 13
    • 0028279349 scopus 로고
    • Expression of a recombinant dystrophin in mdx mice using adenovirus vector
    • Alameddine HS et al. Expression of a recombinant dystrophin in mdx mice using adenovirus vector. Neuromusc Disord 1994; 4: 193-203.
    • (1994) Neuromusc Disord , vol.4 , pp. 193-203
    • Alameddine, H.S.1
  • 14
    • 0028934815 scopus 로고
    • Cellular and humoral immune responses to adeno-viral vectors containing factor IX gene: Tolerization of factor IX and vector antigens allows for long-term expression
    • Dai Y et al. Cellular and humoral immune responses to adeno-viral vectors containing factor IX gene: tolerization of factor IX and vector antigens allows for long-term expression. Proc Natl Acad Sci USA 1995; 92: 1401-1405.
    • (1995) Proc Natl Acad Sci USA , vol.92 , pp. 1401-1405
    • Dai, Y.1
  • 15
    • 0029943155 scopus 로고    scopus 로고
    • A new adenoviral vector: Replacement of all viral coding sequences with 28 kb of DNA independently expressing both full-length dystrophin and β-galactosidase
    • Kochanek S et al. A new adenoviral vector: replacement of all viral coding sequences with 28 kb of DNA independently expressing both full-length dystrophin and β-galactosidase. Proc Natl Acad Sci USA 1996; 93: 5731-5736.
    • (1996) Proc Natl Acad Sci USA , vol.93 , pp. 5731-5736
    • Kochanek, S.1
  • 17
    • 0028032357 scopus 로고
    • Stable delivery of physiologic levels of recombinant erythropoietin to the systemic circulation by intramuscular injection of replication-defective adenovirus
    • Tripathy SK et al Stable delivery of physiologic levels of recombinant erythropoietin to the systemic circulation by intramuscular injection of replication-defective adenovirus. Proc Natl Acad Sci USA 1994; 91. 11557-11561.
    • (1994) Proc Natl Acad Sci USA , vol.91 , pp. 11557-11561
    • Tripathy, S.K.1
  • 18
    • 0028915634 scopus 로고
    • The route of administration is a major determinant of the transduction efficiency of rat tissues by adenoviral recombinants
    • Huard J et al. The route of administration is a major determinant of the transduction efficiency of rat tissues by adenoviral recombinants. Gene Therapy 1995; 2: 107-115.
    • (1995) Gene Therapy , vol.2 , pp. 107-115
    • Huard, J.1
  • 19
    • 0028965811 scopus 로고
    • CNS gene delivery by retrograde transport of recombinant replication-defective adenoviruses
    • Ghadge GD et ai. CNS gene delivery by retrograde transport of recombinant replication-defective adenoviruses. Gene Therapy 1995; 2: 132-137.
    • (1995) Gene Therapy , vol.2 , pp. 132-137
    • Ghadge, G.D.1
  • 20
    • 0028807696 scopus 로고
    • FK506 immunosuppression to control the immune reactions triggered by first-generation adenovirus-mediated gene transfer
    • Vilquin J-T et al. FK506 immunosuppression to control the immune reactions triggered by first-generation adenovirus-mediated gene transfer. Hum Gene Ther 1995; 6: 1391-1401.
    • (1995) Hum Gene Ther , vol.6 , pp. 1391-1401
    • Vilquin, J.-T.1
  • 21
    • 0028328261 scopus 로고
    • Cellular immunity to viral antigens limits El-deleted adenoviruses for gene therapy
    • Yang Y et al. Cellular immunity to viral antigens limits El-deleted adenoviruses for gene therapy. Proc Natl Acad Sci USA 1994; 91: 4407-4411.
    • (1994) Proc Natl Acad Sci USA , vol.91 , pp. 4407-4411
    • Yang, Y.1
  • 22
    • 0026922764 scopus 로고
    • Long-term persistence of plasmid DNA and foreign gene expression in mouse muscle
    • Wolff JA et al. Long-term persistence of plasmid DNA and foreign gene expression in mouse muscle. Hum Mol Genet 1992; 1: 363-369.
    • (1992) Hum Mol Genet , vol.1 , pp. 363-369
    • Wolff, J.A.1
  • 23
    • 0029988465 scopus 로고    scopus 로고
    • Effective treatment of familial hypercholesterolaemia in the mouse model using adenovirus-mediated transfer of the VLDL receptor gene
    • Kozarsky KF et al Effective treatment of familial hypercholesterolaemia in the mouse model using adenovirus-mediated transfer of the VLDL receptor gene. Nat Genet 1996; 13: 54-62.
    • (1996) Nat Genet , vol.13 , pp. 54-62
    • Kozarsky, K.F.1
  • 24
    • 0029936764 scopus 로고    scopus 로고
    • Immune responses to transgene-encoded proteins limit the stability of gene expression after injection of replication-defective adeno-virus vectors
    • Tripathy SK, Black HB, Goldwasser E, Leiden JM. Immune responses to transgene-encoded proteins limit the stability of gene expression after injection of replication-defective adeno-virus vectors. Nature Med 1996; 2: 545-550.
    • (1996) Nature Med , vol.2 , pp. 545-550
    • Tripathy, S.K.1    Black, H.B.2    Goldwasser, E.3    Leiden, J.M.4
  • 25
    • 0023243262 scopus 로고
    • Histochemical staining of clonal mammalian cell lines expressing E. coli β-galactosidase indicate heterogeneous expression of the bacterial gene
    • MacGregor GR, Mogg AE, Burke JF, Caskey CT. Histochemical staining of clonal mammalian cell lines expressing E. coli β-galactosidase indicate heterogeneous expression of the bacterial gene. Somat Cell Mol Genet 1987; 13: 253-265.
    • (1987) Somat Cell Mol Genet , vol.13 , pp. 253-265
    • MacGregor, G.R.1    Mogg, A.E.2    Burke, J.F.3    Caskey, C.T.4
  • 26
    • 0024330387 scopus 로고
    • Cross-reactive protein in Duchenne muscle
    • Hoffman EP et al. Cross-reactive protein in Duchenne muscle Lancet 1989; ii: 1211-1212.
    • (1989) Lancet , vol.2 , pp. 1211-1212
    • Hoffman, E.P.1
  • 27
    • 0025217703 scopus 로고
    • Detailed analysis of the repeat domain of dystrophin reveals four potential hinge segments that may confer flexibility
    • Koenig M, Kunkel LM. Detailed analysis of the repeat domain of dystrophin reveals four potential hinge segments that may confer flexibility. J Biol Chem 1990; 265: 4560-4566.
    • (1990) J Biol Chem , vol.265 , pp. 4560-4566
    • Koenig, M.1    Kunkel, L.M.2
  • 28
    • 0027361264 scopus 로고
    • Primary structure and muscle-specific expression of the 50-kDa dystrophin-associated glycoprotein
    • Roberds SL, Anderson RD, Ibraghimov-Beskrovnaya O, Campbell KP. Primary structure and muscle-specific expression of the 50-kDa dystrophin-associated glycoprotein (adhalin). J Biol Chem 1993; 268: 23739-23742.
    • (1993) J Biol Chem , vol.268 , pp. 23739-23742
    • Roberds, S.L.1    Anderson, R.D.2    Ibraghimov-Beskrovnaya, O.3    Campbell, K.P.4
  • 29
    • 0026543686 scopus 로고
    • Primary structure of dystrophin-associated glycoproteins linking dystrophin to the extracellular matrix
    • Ibraghimov-Beskrovnaya O et al. Primary structure of dystrophin-associated glycoproteins linking dystrophin to the extracellular matrix. Nature 1992; 355: 696-702.
    • (1992) Nature , vol.355 , pp. 696-702
    • Ibraghimov-Beskrovnaya, O.1
  • 30
    • 0027998866 scopus 로고
    • Heterogeneity of the 59-kDa dystrophin-associated protein revealed by cDNA cloning and expression
    • Yang B et al. Heterogeneity of the 59-kDa dystrophin-associated protein revealed by cDNA cloning and expression. J Biol Chem 1994; 269: 6040-6044.
    • (1994) J Biol Chem , vol.269 , pp. 6040-6044
    • Yang, B.1


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