-
1
-
-
0038644348
-
-
(ed) Oxford University Press Inc.: New York, Oxford
-
Emery AEH (ed). The Muscular Dystrophies. Oxford University Press Inc.: New York, Oxford, 2001.
-
(2001)
The Muscular Dystrophies
-
-
Emery, A.E.H.1
-
2
-
-
0042170127
-
Gene therapy for muscular dystrophy - A review of promising progress
-
Gregorevic P, Chamberlain JS. Gene therapy for muscular dystrophy - a review of promising progress. Expert Opin Biol Ther 2003; 3: 803-814.
-
(2003)
Expert Opin. Biol. Ther.
, vol.3
, pp. 803-814
-
-
Gregorevic, P.1
Chamberlain, J.S.2
-
3
-
-
0027186053
-
Overexpression of dystrophin in transgenic mdx mice eliminates dystrophic symptoms without toxicity
-
Cox GA et al. Overexpression of dystrophin in transgenic mdx mice eliminates dystrophic symptoms without toxicity. Nature 1993; 364: 725-729.
-
(1993)
Nature
, vol.364
, pp. 725-729
-
-
Cox, G.A.1
-
4
-
-
0036127393
-
Modular flexibility of dystrophin: Implications for gene therapy of Duchenne muscular dystrophy
-
Harper SQ et al. Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy. Nat Med 2002; 8: 253-261.
-
(2002)
Nat. Med.
, vol.8
, pp. 253-261
-
-
Harper, S.Q.1
-
5
-
-
0036790998
-
Functional correction of adult mdx mouse muscle using gutted adenoviral vectors expressing full-length dystrophin
-
DelloRusso C et al. Functional correction of adult mdx mouse muscle using gutted adenoviral vectors expressing full-length dystrophin. Proc Natl Acad Sci USA 2002; 99: 12979-12984.
-
(2002)
Proc. Natl. Acad. Sci. USA
, vol.99
, pp. 12979-12984
-
-
DelloRusso, C.1
-
6
-
-
4043092073
-
Systemic delivery of genes to striated muscles using adeno-associated viral vectors
-
Gregorevic P et al. Systemic delivery of genes to striated muscles using adeno-associated viral vectors. Nat Med 2004; 10: 828-834.
-
(2004)
Nat. Med.
, vol.10
, pp. 828-834
-
-
Gregorevic, P.1
-
7
-
-
0033023881
-
Structure of adeno-associated virus vector DNA following transduction of the skeletal muscle
-
Vincent-Lacaze N et al. Structure of adeno-associated virus vector DNA following transduction of the skeletal muscle. J Virol 1999; 73: 1949-1955.
-
(1999)
J. Virol.
, vol.73
, pp. 1949-1955
-
-
Vincent-Lacaze, N.1
-
8
-
-
0037333815
-
Genetic fate of recombinant adeno-associated virus vector genomes in muscle
-
Schnepp BC et al. Genetic fate of recombinant adeno-associated virus vector genomes in muscle. J Virol 2003; 77: 3495-3504.
-
(2003)
J. Virol.
, vol.77
, pp. 3495-3504
-
-
Schnepp, B.C.1
-
9
-
-
0029843596
-
Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector
-
Xiao X, Li J, Samulski RJ. Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. J Virol 1996; 70: 8098-8108.
-
(1996)
J. Virol.
, vol.70
, pp. 8098-8108
-
-
Xiao, X.1
Li, J.2
Samulski, R.J.3
-
10
-
-
0030671462
-
Efficient and stable adeno-associated virus-mediated transduction in the skeletal muscle of adult immunocompetent mice
-
Snyder RO et al. Efficient and stable adeno-associated virus-mediated transduction in the skeletal muscle of adult immunocompetent mice. Hum Gene Ther 1997; 8: 1891-1900.
-
(1997)
Hum. Gene Ther.
, vol.8
, pp. 1891-1900
-
-
Snyder, R.O.1
-
11
-
-
0033056699
-
Frequency and stability of chromosomal integration of adenovirus vectors
-
Harui A, Suzuki S, Kochanek S, Mitani K. Frequency and stability of chromosomal integration of adenovirus vectors. J Virol 1999; 73: 6141-6146.
-
(1999)
J. Virol.
, vol.73
, pp. 6141-6146
-
-
Harui, A.1
Suzuki, S.2
Kochanek, S.3
Mitani, K.4
-
12
-
-
0030716959
-
Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors
-
Kafri T et al. Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors. Nat Genet 1997; 17: 314-317.
-
(1997)
Nat. Genet.
, vol.17
, pp. 314-317
-
-
Kafri, T.1
-
13
-
-
0035437193
-
Lentivirus administration to rat muscle provides efficient sustained expression of erythropoietin
-
Seppen J, Barry SC, Harder B, Osborne WR. Lentivirus administration to rat muscle provides efficient sustained expression of erythropoietin. Blood 2001; 98: 594-596.
-
(2001)
Blood
, vol.98
, pp. 594-596
-
-
Seppen, J.1
Barry, S.C.2
Harder, B.3
Osborne, W.R.4
-
14
-
-
0346035738
-
Efficient transduction of liver and muscle after in utero injection of lentiviral vectors with different pseudotypes
-
MacKenzie TC et al. Efficient transduction of liver and muscle after in utero injection of lentiviral vectors with different pseudotypes. Mol Ther 2002; 6: 349-358.
-
(2002)
Mol. Ther.
, vol.6
, pp. 349-358
-
-
MacKenzie, T.C.1
-
15
-
-
0142042481
-
Correction of the dystrophic phenotype by in vivo targeting of muscle progenitor cells
-
Kobinger GP et al. Correction of the dystrophic phenotype by in vivo targeting of muscle progenitor cells. Hum Gene Ther 2003; 14: 1441-1449.
-
(2003)
Hum. Gene Ther.
, vol.14
, pp. 1441-1449
-
-
Kobinger, G.P.1
-
16
-
-
0002841587
-
The satellite cell and muscle regeneration
-
Andrew G, Engel CF-A (eds). McGraw-Hill Inc
-
Bischoff R. The satellite cell and muscle regeneration. In: Andrew G, Engel CF-A (eds). Myology. McGraw-Hill Inc., 1994: pp 97-118.
-
(1994)
Myology
, pp. 97-118
-
-
Bischoff, R.1
-
17
-
-
0033598374
-
Dystrophin expression in the mdx mouse restored by stem cell transplantation
-
Gussoni E et al. Dystrophin expression in the mdx mouse restored by stem cell transplantation. Nature 1999; 401: 390-394.
-
(1999)
Nature
, vol.401
, pp. 390-394
-
-
Gussoni, E.1
-
18
-
-
0035831648
-
Flow cytometric characterization of myogenic cell populations obtained via the preplate technique: Potential for rapid isolation of muscle-derived stem cells
-
Jankowski RJ, Haluszczak C, Trucco M, Huard J. Flow cytometric characterization of myogenic cell populations obtained via the preplate technique: potential for rapid isolation of muscle-derived stem cells. Hum Gene Ther 2001; 12: 619-628.
-
(2001)
Hum. Gene Ther.
, vol.12
, pp. 619-628
-
-
Jankowski, R.J.1
Haluszczak, C.2
Trucco, M.3
Huard, J.4
-
19
-
-
0036322544
-
Multipotent progenitor cells can be isolated from postnatal murine bone marrow, muscle, and brain
-
Jiang Y et al. Multipotent progenitor cells can be isolated from postnatal murine bone marrow, muscle, and brain. Exp Hematol 2002; 30: 896-904.
-
(2002)
Exp. Hematol.
, vol.30
, pp. 896-904
-
-
Jiang, Y.1
-
20
-
-
0036980730
-
Autotransplantation in mdx mice of mdx myoblasts genetically corrected by an HSV-1 amplicon vector
-
Bujold M et al. Autotransplantation in mdx mice of mdx myoblasts genetically corrected by an HSV-1 amplicon vector. Cell Transplant 2002; 11: 759-767.
-
(2002)
Cell Transplant.
, vol.11
, pp. 759-767
-
-
Bujold, M.1
-
21
-
-
6844257545
-
Ex vivo gene transfer using adenovirus-mediated full-length dystrophin delivery to dystrophic muscles
-
Floyd Jr SS et al. Ex vivo gene transfer using adenovirus-mediated full-length dystrophin delivery to dystrophic muscles. Gene Therapy 1998; 5: 19-30.
-
(1998)
Gene Therapy
, vol.5
, pp. 19-30
-
-
Floyd Jr., S.S.1
-
22
-
-
0031788860
-
Expression of human dystrophin following the transplantation of genetically modified mdx myoblasts
-
Moisset PA, Gagnon Y, Karpati G, Tremblay JP. Expression of human dystrophin following the transplantation of genetically modified mdx myoblasts. Gene Therapy 1998; 5: 1340-1346.
-
(1998)
Gene Therapy
, vol.5
, pp. 1340-1346
-
-
Moisset, P.A.1
Gagnon, Y.2
Karpati, G.3
Tremblay, J.P.4
-
23
-
-
0035667634
-
Immune evasion by muscle-specific gene expression in dystrophic muscle
-
Hartigan-O'Connor D et al. Immune evasion by muscle-specific gene expression in dystrophic muscle. Mol Ther 2001; 4: 525-533.
-
(2001)
Mol. Ther.
, vol.4
, pp. 525-533
-
-
Hartigan-O'Connor, D.1
-
24
-
-
0035915778
-
Muscle-specific promoters may be necessary for adeno-associated virus-mediated gene transfer in the treatment of muscular dystrophies
-
Cordier L et al. Muscle-specific promoters may be necessary for adeno-associated virus-mediated gene transfer in the treatment of muscular dystrophies. Hum Gene Ther 2001; 12: 205-215.
-
(2001)
Hum. Gene Ther.
, vol.12
, pp. 205-215
-
-
Cordier, L.1
-
25
-
-
0034218590
-
Analysis of muscle creatine kinase regulatory elements in recombinant adenoviral vectors
-
Hauser MA et al. Analysis of muscle creatine kinase regulatory elements in recombinant adenoviral vectors. Mol Ther 2000; 2: 16-25.
-
(2000)
Mol. Ther.
, vol.2
, pp. 16-25
-
-
Hauser, M.A.1
-
26
-
-
0030634743
-
Skeletal muscle cultures
-
Neville C et al. Skeletal muscle cultures. Methods Cell Biol 1997; 52: 85-116.
-
(1997)
Methods Cell Biol.
, vol.52
, pp. 85-116
-
-
Neville, C.1
-
27
-
-
0023606959
-
Growth factor control of skeletal muscle differentiation: Commitment to terminal differentiation occurs in G1 phase and is repressed by fibroblast growth factor
-
Clegg CH, Linkhart TA, Olwin BB, Hauschka SD. Growth factor control of skeletal muscle differentiation: commitment to terminal differentiation occurs in G1 phase and is repressed by fibroblast growth factor. J Cell Biol 1987; 105: 949-956.
-
(1987)
J. Cell Biol.
, vol.105
, pp. 949-956
-
-
Clegg, C.H.1
Linkhart, T.A.2
Olwin, B.B.3
Hauschka, S.D.4
-
28
-
-
0021927243
-
Regulation of creatine kinase induction in differentiating mouse myoblasts
-
Chamberlain JS, Jaynes JB, Hauschka SD. Regulation of creatine kinase induction in differentiating mouse myoblasts. Mol Cell Biol 1985; 5: 484-492.
-
(1985)
Mol. Cell Biol.
, vol.5
, pp. 484-492
-
-
Chamberlain, J.S.1
Jaynes, J.B.2
Hauschka, S.D.3
-
29
-
-
0022767343
-
Transcriptional regulation of the muscle creatine kinase gene and regulated expression in transfected mouse myoblasts
-
Jaynes JB et al. Transcriptional regulation of the muscle creatine kinase gene and regulated expression in transfected mouse myoblasts. Mol Cell Biol 1986; 6: 2855-2864.
-
(1986)
Mol. Cell Biol.
, vol.6
, pp. 2855-2864
-
-
Jaynes, J.B.1
-
30
-
-
0032191380
-
Lentiviruses as gene transfer agents for delivery to non-dividing cells
-
Naldini L. Lentiviruses as gene transfer agents for delivery to non-dividing cells. Curr Opin Biotechnol 1998; 9: 457-463.
-
(1998)
Curr. Opin. Biotechnol.
, vol.9
, pp. 457-463
-
-
Naldini, L.1
-
31
-
-
0036664451
-
Highly efficient lentiviral vector-mediated transduction of nondividing, fully reimplantable primary hepatocytes
-
Nguyen TH et al. Highly efficient lentiviral vector-mediated transduction of nondividing, fully reimplantable primary hepatocytes. Mol Ther 2002; 6: 199-209.
-
(2002)
Mol. Ther.
, vol.6
, pp. 199-209
-
-
Nguyen, T.H.1
-
32
-
-
0038742916
-
'Advanced' generation lentiviruses as efficient vectors for cardiomyocyte gene transduction in vitro and in vivo
-
Bonci D et al. 'Advanced' generation lentiviruses as efficient vectors for cardiomyocyte gene transduction in vitro and in vivo. Gene Therapy 2003; 10: 630-636.
-
(2003)
Gene Therapy
, vol.10
, pp. 630-636
-
-
Bonci, D.1
-
33
-
-
0029993858
-
Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector
-
Naldini L et al. Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector. Proc Natl Acad Sci USA 1996; 93: 11382-11388.
-
(1996)
Proc. Natl. Acad. Sci. USA
, vol.93
, pp. 11382-11388
-
-
Naldini, L.1
-
34
-
-
0033831002
-
Lentiviral vector transduction of hematopoietic stem cells that mediate long-term reconstitution of lethally irradiated mice
-
Chen W et al. Lentiviral vector transduction of hematopoietic stem cells that mediate long-term reconstitution of lethally irradiated mice. Stem Cells 2000; 18: 352-359.
-
(2000)
Stem Cells
, vol.18
, pp. 352-359
-
-
Chen, W.1
-
35
-
-
0042424676
-
Skeletal muscle engraftment potential of adult mouse skin side population cells
-
Montanaro F et al. Skeletal muscle engraftment potential of adult mouse skin side population cells. Proc Natl Acad Sci USA 2003; 100: 9336-9341.
-
(2003)
Proc. Natl. Acad. Sci. USA
, vol.100
, pp. 9336-9341
-
-
Montanaro, F.1
-
36
-
-
1542513708
-
Systemic delivery of human microdystrophin to regenerating mouse dystrophic muscle by muscle progenitor cells
-
Bachrach E et al. Systemic delivery of human microdystrophin to regenerating mouse dystrophic muscle by muscle progenitor cells. Proc Natl Acad Sci USA 2004; 101: 3581-3586.
-
(2004)
Proc. Natl. Acad. Sci. USA
, vol.101
, pp. 3581-3586
-
-
Bachrach, E.1
-
37
-
-
0028346944
-
A differential efficiency of adenovirus-mediated in vivo gene transfer into skeletal muscle cells of different maturity
-
Acsadi G et al. A differential efficiency of adenovirus-mediated in vivo gene transfer into skeletal muscle cells of different maturity. Hum Mol Genet 1994; 3: 579-584.
-
(1994)
Hum. Mol. Genet.
, vol.3
, pp. 579-584
-
-
Acsadi, G.1
-
38
-
-
0033541587
-
Expression of the primary coxsackie and adenovirus receptor is downregulated during skeletal muscle maturation and limits the efficacy of adenovirus-mediated gene delivery to muscle cells
-
Nalbantoglu J, Pari G, Karpati G, Holland PC. Expression of the primary coxsackie and adenovirus receptor is downregulated during skeletal muscle maturation and limits the efficacy of adenovirus-mediated gene delivery to muscle cells. Hum Gene Ther 1999; 10: 1009-1019.
-
(1999)
Hum. Gene Ther.
, vol.10
, pp. 1009-1019
-
-
Nalbantoglu, J.1
Pari, G.2
Karpati, G.3
Holland, P.C.4
-
39
-
-
0141613885
-
Fetal muscle gene transfer is not enhanced by an RGD capsid modification to high-capacity adenoviral vectors
-
Bilbao R et al. Fetal muscle gene transfer is not enhanced by an RGD capsid modification to high-capacity adenoviral vectors. Gene Therapy 2003; 10: 1821-1829.
-
(2003)
Gene Therapy
, vol.10
, pp. 1821-1829
-
-
Bilbao, R.1
-
40
-
-
0036112584
-
Cell surface heparan sulfate is a receptor for attachment of envelope protein-free retrovirus-like particles and VSV-G pseudotyped MLV-derived retrovirus vectors to target cells
-
Guibinga GH, Miyanohara A, Esko JD, Friedmann T. Cell surface heparan sulfate is a receptor for attachment of envelope protein-free retrovirus-like particles and VSV-G pseudotyped MLV-derived retrovirus vectors to target cells. Mol Ther 2002; 5: 538-546.
-
(2002)
Mol. Ther.
, vol.5
, pp. 538-546
-
-
Guibinga, G.H.1
Miyanohara, A.2
Esko, J.D.3
Friedmann, T.4
-
42
-
-
0003604683
-
-
ASM press: Washington DC
-
Flint SJ, Enquist LW, Krug RM, Racaniello VR, Skalka AM. Principles of Virology, Molecular Biology, Pathogenesis, and Control. ASM press: Washington DC, 2000.
-
(2000)
Principles of Virology, Molecular Biology, Pathogenesis, and Control
-
-
Flint, S.J.1
Enquist, L.W.2
Krug, R.M.3
Racaniello, V.R.4
Skalka, A.M.5
-
43
-
-
0036957811
-
Efficient gene transfer into the CNS by lentiviral vectors purified by anion exchange chromatography
-
Scherr M et al. Efficient gene transfer into the CNS by lentiviral vectors purified by anion exchange chromatography. Gene Therapy 2002; 9: 1708-1714.
-
(2002)
Gene Therapy
, vol.9
, pp. 1708-1714
-
-
Scherr, M.1
-
44
-
-
0036370361
-
Generation of HIV-1 derived lentiviral vectors
-
Follenzi A, Naldini L. Generation of HIV-1 derived lentiviral vectors. Methods Enzymol 2002; 346: 454-465.
-
(2002)
Methods Enzymol.
, vol.346
, pp. 454-465
-
-
Follenzi, A.1
Naldini, L.2
-
46
-
-
0036366386
-
Streamlined large-scale production of recombinant adeno-associated virus (rAAV) vectors
-
Potter M et al. Streamlined large-scale production of recombinant adeno-associated virus (rAAV) vectors. Methods Enzymol 2002; 346: 413-430.
-
(2002)
Methods Enzymol.
, vol.346
, pp. 413-430
-
-
Potter, M.1
-
47
-
-
4644252083
-
Efficient transduction of skeletal muscle using vectors based on adeno-associated virus serotype 6
-
Blankinship MJ et al. Efficient transduction of skeletal muscle using vectors based on adeno-associated virus serotype 6. Mol Ther 2004; 10: 671-678.
-
(2004)
Mol. Ther.
, vol.10
, pp. 671-678
-
-
Blankinship, M.J.1
-
48
-
-
0034919136
-
Lentivirus vectors encoding both central polypurine tract and posttranscriptional regulatory element provide enhanced transduction and transgene expression
-
Barry SC et al. Lentivirus vectors encoding both central polypurine tract and posttranscriptional regulatory element provide enhanced transduction and transgene expression. Hum Gene Ther 2001; 12: 1103-1108.
-
(2001)
Hum. Gene Ther.
, vol.12
, pp. 1103-1108
-
-
Barry, S.C.1
-
49
-
-
0028385758
-
Versatile retroviral vectors for potential use in gene therapy
-
Hawley RG, Lieu FH, Fong AZ, Hawley TS. Versatile retroviral vectors for potential use in gene therapy. Gene Therapy 1994; 1: 136-138.
-
(1994)
Gene Therapy
, vol.1
, pp. 136-138
-
-
Hawley, R.G.1
Lieu, F.H.2
Fong, A.Z.3
Hawley, T.S.4
-
50
-
-
0022367618
-
Slow and fast myosin heavy chain content defines three types of myotubes in early muscle cell cultures
-
Miller JB, Crow MT, Stockdale FE. Slow and fast myosin heavy chain content defines three types of myotubes in early muscle cell cultures. J Cell Biol 1985; 101: 1643-1650.
-
(1985)
J. Cell Biol.
, vol.101
, pp. 1643-1650
-
-
Miller, J.B.1
Crow, M.T.2
Stockdale, F.E.3
|