-
1
-
-
79960549659
-
-
Muscle Nerve (this issue)
-
Zimmerman A, Clemens PR, Tesi-Rocha C, Connolly A, Iannaccone ST, Kuntz N, et al. Liquid formulation of pentoxifylline is a poorly tolerated treatment for Duchenne dystrophy. Muscle Nerve (this issue).
-
Liquid Formulation of Pentoxifylline is A Poorly Tolerated Treatment For Duchenne Dystrophy
-
-
Zimmerman, A.1
Clemens, P.R.2
Tesi-Rocha, C.3
Connolly, A.4
Iannaccone, S.T.5
Kuntz, N.6
-
4
-
-
0024094087
-
Localization of the mdx mutation within the mouse dystrophin gene
-
Ryder-Cook AS, Sicinski P, Thomas K, Davies KE, Worton RG, Barnard EA, et al. Localization of the mdx mutation within the mouse dystrophin gene. EMBO J 1988;7: 3017-3021.
-
(1988)
EMBO J
, vol.7
, pp. 3017-3021
-
-
Ryder-Cook, A.S.1
Sicinski, P.2
Thomas, K.3
Davies, K.E.4
Worton, R.G.5
Barnard, E.A.6
-
5
-
-
0024353559
-
The molecular basis of muscular dystrophy in the mdx mouse: A point mutation
-
Sicinski P, Geng Y, Ryder-Cook AS, Barnard EA, Darlison MG, Barnard PJ. The molecular basis of muscular dystrophy in the mdx mouse: a point mutation. Science 1989;244: 1578-1580.
-
(1989)
Science
, vol.244
, pp. 1578-1580
-
-
Sicinski, P.1
Geng, Y.2
Ryder-Cook, A.S.3
Barnard, E.A.4
Darlison, M.G.5
Barnard, P.J.6
-
6
-
-
0036591684
-
Muscular dystrophies involving the dystrophin-glycoprotein complex: An overview of current mouse models
-
Durbeej M, Campbell KP. Muscular dystrophies involving the dystrophin-glycoprotein complex: an overview of current mouse models. Curr Opin Genet Dev 2002;12:349-361.
-
(2002)
Curr Opin Genet Dev
, vol.12
, pp. 349-361
-
-
Durbeej, M.1
Campbell, K.P.2
-
7
-
-
0030848969
-
Utrophin-dystrophin-deficient mice as a model for Duchenne muscular dystrophy
-
Deconinck AE, Rafael JA, Skinner JA, Brown SC, Potter AC, Metzinger L, et al. Utrophin-dystrophin-deficient mice as a model for Duchenne muscular dystrophy. Cell 1997;90: 717-727.
-
(1997)
Cell
, vol.90
, pp. 717-727
-
-
Deconinck, A.E.1
Rafael, J.A.2
Skinner, J.A.3
Brown, S.C.4
Potter, A.C.5
Metzinger, L.6
-
8
-
-
0030848338
-
Skeletal and cardiac myopathies in mice lacking utrophin and dystrophin: A model for Duchenne muscular dystrophy
-
Grady RM, Teng H, Nichol MC, Cunningham JC, Wilkinson RS, Sanes JR. Skeletal and cardiac myopathies in mice lacking utrophin and dystrophin: a model for Duchenne muscular dystrophy. Cell 1997;90:729-738.
-
(1997)
Cell
, vol.90
, pp. 729-738
-
-
Grady, R.M.1
Teng, H.2
Nichol, M.C.3
Cunningham, J.C.4
Wilkinson, R.S.5
Sanes, J.R.6
-
10
-
-
0034812094
-
Dystrophin in adult zebrafish muscle
-
Chambers SP, Dodd A, Overall R, Sirey T, Lam LT, Morris GE, Love DR. Dystrophin in adult zebrafish muscle. Biochem Biophys Res Commun 2001;286:478-483.
-
(2001)
Biochem Biophys Res Commun
, vol.286
, pp. 478-483
-
-
Chambers, S.P.1
Dodd, A.2
Overall, R.3
Sirey, T.4
Lam, L.T.5
Morris, G.E.6
Love, D.R.7
-
11
-
-
0037444335
-
Dystrophin associated protein complex in zebrafish
-
Guyon JR, Mosley AN, Zhou Y, O'Brien KF, Sheng X, Chiang K, et al. Dystrophin associated protein complex in zebrafish. Hum Mol Genet 2003;12:601-615.
-
(2003)
Hum Mol Genet
, vol.12
, pp. 601-615
-
-
Guyon, J.R.1
Mosley, A.N.2
Zhou, Y.3
O'Brien, K.F.4
Sheng, X.5
Chiang, K.6
-
12
-
-
34147188075
-
Zebrafish orthologs of human muscular dystrophy genes
-
Steffen LS, Guyon JR, Vogel ED, Beltre R, Pusack TJ, Zhou Y, et al. Zebrafish orthologs of human muscular dystrophy genes. BMC Genomics 2007;8:79.
-
(2007)
BMC Genomics
, vol.8
, pp. 79
-
-
Steffen, L.S.1
Guyon, J.R.2
Vogel, E.D.3
Beltre, R.4
Pusack, T.J.5
Zhou, Y.6
-
13
-
-
78650170393
-
Dystrophin-deficient zebrafish feature aspects of the Duchenne muscular dystrophy pathology
-
Berger J, Berger S, Hall TE, Lieschke GJ, Currie PD. Dystrophin-deficient zebrafish feature aspects of the Duchenne muscular dystrophy pathology. Neuromuscul Disord 2010; 20:826-832.
-
(2010)
Neuromuscul Disord
, vol.20
, pp. 826-832
-
-
Berger, J.1
Berger, S.2
Hall, T.E.3
Lieschke, G.J.4
Currie, P.D.5
-
14
-
-
57649193108
-
Genetic isolation and characterization of a splicing mutant of zebrafish dystrophin
-
Guyon JR, Goswami J, Jun SJ, Thorne M, Howell M, Pusack T, et al. Genetic isolation and characterization of a splicing mutant of zebrafish dystrophin. Hum Mol Genet 2009;18: 202-211.
-
(2009)
Hum Mol Genet
, vol.18
, pp. 202-211
-
-
Guyon, J.R.1
Goswami, J.2
Jun, S.J.3
Thorne, M.4
Howell, M.5
Pusack, T.6
-
15
-
-
0036637659
-
Removal of dystroglycan causes severe muscular dystrophy in zebrafish embryos
-
Parsons MJ, Campos I, Hirst EM, Stemple DL. Removal of dystroglycan causes severe muscular dystrophy in zebrafish embryos. Development 2002;129:3505-3512.
-
(2002)
Development
, vol.129
, pp. 3505-3512
-
-
Parsons, M.J.1
Campos, I.2
Hirst, E.M.3
Stemple, D.L.4
-
16
-
-
13544251544
-
Delta-sarcoglycan is required for early zebrafish muscle organization
-
Guyon JR, Mosley AN, Jun SJ, Montanaro F, Steffen LS, Zhou Y, et al. Delta-sarcoglycan is required for early zebrafish muscle organization. Exp Cell Res 2005;304:105-115.
-
(2005)
Exp Cell Res
, vol.304
, pp. 105-115
-
-
Guyon, J.R.1
Mosley, A.N.2
Jun, S.J.3
Montanaro, F.4
Steffen, L.S.5
Zhou, Y.6
-
18
-
-
0023883621
-
The homologue of the Duchenne locus is defective in X-linked muscular dystrophy of dogs
-
Cooper BJ, Winand NJ, Stedman H, Valentine BA, Hoffman EP, Kunkel LM, et al. The homologue of the Duchenne locus is defective in X-linked muscular dystrophy of dogs. Nature 1988;334:154-156.
-
(1988)
Nature
, vol.334
, pp. 154-156
-
-
Cooper, B.J.1
Winand, N.J.2
Stedman, H.3
Valentine, B.A.4
Hoffman, E.P.5
Kunkel, L.M.6
-
19
-
-
0026538578
-
An error in dystrophin mRNA processing in golden retriever muscular dystrophy, an animal homologue of Duchenne muscular dystrophy
-
Sharp NJ, Kornegay JN, van Camp SD, Herbstreith MH, Secore SL, Kettle S, et al. An error in dystrophin mRNA processing in golden retriever muscular dystrophy, an animal homologue of Duchenne muscular dystrophy. Genomics 1992;13:115-121.
-
(1992)
Genomics
, vol.13
, pp. 115-121
-
-
Sharp, N.J.1
Kornegay, J.N.2
van Camp, S.D.3
Herbstreith, M.H.4
Secore, S.L.5
Kettle, S.6
-
20
-
-
0024540879
-
Conversion of mdx myofibres from dystrophin-negative to -positive by injection of normal myoblasts
-
Partridge TA, Morgan JE, Coulton GR, Hoffman EP, Kunkel LM. Conversion of mdx myofibres from dystrophin-negative to -positive by injection of normal myoblasts. Nature 1989; 337:176-179.
-
(1989)
Nature
, vol.337
, pp. 176-179
-
-
Partridge, T.A.1
Morgan, J.E.2
Coulton, G.R.3
Hoffman, E.P.4
Kunkel, L.M.5
-
21
-
-
0026560260
-
Normal dystrophin transcripts detected in Duchenne muscular dystrophy patients after myoblast transplantation
-
Gussoni E, Pavlath GK, Lanctot AM, Sharma KR, Miller RG, Steinman L, et al. Normal dystrophin transcripts detected in Duchenne muscular dystrophy patients after myoblast transplantation. Nature 1992;356:435-438.
-
(1992)
Nature
, vol.356
, pp. 435-438
-
-
Gussoni, E.1
Pavlath, G.K.2
Lanctot, A.M.3
Sharma, K.R.4
Miller, R.G.5
Steinman, L.6
-
22
-
-
0026593840
-
Human myoblast transplantation: Preliminary results of 4 cases
-
Huard J, Bouchard JP, Roy R, Malouin F, Dansereau G, Labrecque C, et al. Human myoblast transplantation: preliminary results of 4 cases. Muscle Nerve 1992;15:550-560.
-
(1992)
Muscle Nerve
, vol.15
, pp. 550-560
-
-
Huard, J.1
Bouchard, J.P.2
Roy, R.3
Malouin, F.4
Dansereau, G.5
Labrecque, C.6
-
23
-
-
0027269957
-
Myoblast transfer in Duchenne muscular dystrophy
-
Karpati G, Ajdukovic D, Arnold D, Gledhill RB, Guttmann R, Holland P, et al. Myoblast transfer in Duchenne muscular dystrophy. Ann Neurol 1993;34:8-17.
-
(1993)
Ann Neurol
, vol.34
, pp. 8-17
-
-
Karpati, G.1
Ajdukovic, D.2
Arnold, D.3
Gledhill, R.B.4
Guttmann, R.5
Holland, P.6
-
24
-
-
0029129834
-
Myoblast transfer in the treatment of Duchenne's muscular dystrophy
-
Mendell JR, Kissel JT, Amato AA, King W, Signore L, Prior TW, et al. Myoblast transfer in the treatment of Duchenne's muscular dystrophy. N Engl J Med 1995;333:832-838.
-
(1995)
N Engl J Med
, vol.333
, pp. 832-838
-
-
Mendell, J.R.1
Kissel, J.T.2
Amato, A.A.3
King, W.4
Signore, L.5
Prior, T.W.6
-
25
-
-
8244228688
-
Myoblast implantation in Duchenne muscular dystrophy: The San Francisco study
-
Miller RG, Sharma KR, Pavlath GK, Gussoni E, Mynhier M, Lanctot AM, et al. Myoblast implantation in Duchenne muscular dystrophy: the San Francisco study. Muscle Nerve 1997;20:469-478.
-
(1997)
Muscle Nerve
, vol.20
, pp. 469-478
-
-
Miller, R.G.1
Sharma, K.R.2
Pavlath, G.K.3
Gussoni, E.4
Mynhier, M.5
Lanctot, A.M.6
-
26
-
-
0027721951
-
Myoblast transplantation between monozygotic twin girl carriers of Duchenne muscular dystrophy
-
Tremblay JP, Bouchard JP, Malouin F, Theau D, Cottrell F, Collin H, et al. Myoblast transplantation between monozygotic twin girl carriers of Duchenne muscular dystrophy. Neuromuscul Disord 1993;3:583-592.
-
(1993)
Neuromuscul Disord
, vol.3
, pp. 583-592
-
-
Tremblay, J.P.1
Bouchard, J.P.2
Malouin, F.3
Theau, D.4
Cottrell, F.5
Collin, H.6
-
27
-
-
62849120577
-
Blastocyst injection of wild type embryonic stem cells induces global corrections in mdx mice
-
Stillwell E, Vitale J, Zhao Q, Beck A, Schneider J, Khadim F, et al. Blastocyst injection of wild type embryonic stem cells induces global corrections in mdx mice. PLoS One 2009;4: e4759.
-
(2009)
PLoS One
, vol.4
-
-
Stillwell, E.1
Vitale, J.2
Zhao, Q.3
Beck, A.4
Schneider, J.5
Khadim, F.6
-
28
-
-
0034610364
-
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 USA 2000;97:13714-13719.
-
(2000)
Proc Natl Acad Sci USA
, vol.97
, pp. 13714-13719
-
-
Wang, B.1
Li, J.2
Xiao, X.3
-
29
-
-
27944479711
-
Efficient in vivo gene expression by trans-splicing adenoassociated viral vectors
-
Lai Y, Yue Y, Liu M, Ghosh A, Engelhardt JF, Chamberlain JS, et al. Efficient in vivo gene expression by trans-splicing adenoassociated viral vectors. Nat Biotechnol 2005;23:1435-1439.
-
(2005)
Nat Biotechnol
, vol.23
, pp. 1435-1439
-
-
Lai, Y.1
Yue, Y.2
Liu, M.3
Ghosh, A.4
Engelhardt, J.F.5
Chamberlain, J.S.6
-
30
-
-
7044253593
-
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
-
31
-
-
4043092073
-
Systemic delivery of genes to striated muscles using adeno-associated viral vectors
-
Gregorevic P, Blankinship MJ, Allen JM, Crawford RW, Meuse L, Miller DG, 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
Blankinship, M.J.2
Allen, J.M.3
Crawford, R.W.4
Meuse, L.5
Miller, D.G.6
-
32
-
-
12744269885
-
Adeno-associated virus-mediated microdystrophin expression protects young mdx muscle from contractioninduced injury
-
Liu M, Yue Y, Harper SQ, Grange RW, Chamberlain JS, Duan D. Adeno-associated virus-mediated microdystrophin expression protects young mdx muscle from contractioninduced 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
-
33
-
-
50549098032
-
Microutrophin delivery through rAAV6 increases lifespan and improves muscle function in dystrophic dystrophin/ utrophin-deficient mice
-
Odom GL, Gregorevic P, Allen JM, Finn E, Chamberlain JS. Microutrophin delivery through rAAV6 increases lifespan and improves muscle function in dystrophic dystrophin/ utrophin-deficient mice. Mol Ther 2008;16:1539-1545.
-
(2008)
Mol Ther
, vol.16
, pp. 1539-1545
-
-
Odom, G.L.1
Gregorevic, P.2
Allen, J.M.3
Finn, E.4
Chamberlain, J.S.5
-
34
-
-
77957725001
-
Dystrophin immunity in Duchenne's muscular dystrophy
-
Mendell JR, Campbell K, Rodino-Klapac L, Sahenk Z, Shilling C, Lewis S, et al. Dystrophin immunity in Duchenne's muscular dystrophy. N Engl J Med 2010;363:1429-1437.
-
(2010)
N Engl J Med
, vol.363
, pp. 1429-1437
-
-
Mendell, J.R.1
Campbell, K.2
Rodino-Klapac, L.3
Sahenk, Z.4
Shilling, C.5
Lewis, S.6
-
35
-
-
69949107887
-
Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: A single-blind, placebo-controlled, doseescalation, proof-of-concept study
-
Kinali M, Arechavala-Gomeza V, Feng L, Cirak S, Hunt D, Adkin C, et al. Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, doseescalation, proof-of-concept study. Lancet Neurol 2009;8: 918-928.
-
(2009)
Lancet Neurol
, vol.8
, pp. 918-928
-
-
Kinali, M.1
Arechavala-Gomeza, V.2
Feng, L.3
Cirak, S.4
Hunt, D.5
Adkin, C.6
-
36
-
-
37549034298
-
Local dystrophin restoration with antisense oligonucleotide PRO051
-
van Deutekom JC, Janson AA, Ginjaar IB, Frankhuizen WS, Aartsma-Rus A, Bremmer-Bout M, et al. Local dystrophin restoration with antisense oligonucleotide PRO051. N Engl J Med 2007;357:2677-2686.
-
(2007)
N Engl J Med
, vol.357
, pp. 2677-2686
-
-
van Deutekom, J.C.1
Janson, A.A.2
Ginjaar, I.B.3
Frankhuizen, W.S.4
Aartsma-Rus, A.5
Bremmer-Bout, M.6
-
37
-
-
44849140764
-
A phase I/II trial of MYO-029 in adult subjects with muscular dystrophy
-
Wagner KR, Fleckenstein JL, Amato AA, Barohn RJ, Bushby K, Escolar DM, et al. A phase I/II trial of MYO-029 in adult subjects with muscular dystrophy. Ann Neurol 2008;63: 561-571.
-
(2008)
Ann Neurol
, vol.63
, pp. 561-571
-
-
Wagner, K.R.1
Fleckenstein, J.L.2
Amato, A.A.3
Barohn, R.J.4
Bushby, K.5
Escolar, D.M.6
|