-
1
-
-
0036127393
-
Modular flexibility of dystrophin: Implications for gene therapy of Duchenne muscular dystrophy
-
Harper, SQ, Hauser, MA, DelloRusso, C, Duan, D, Crawford, RW, Phelps, SF et al. (2002). Modular flexibility of dystrophin: Implications for gene therapy of Duchenne muscular dystrophy. Nat Med 8: 253-261.
-
(2002)
Nat Med
, vol.8
, pp. 253-261
-
-
Harper, S.Q.1
Hauser, M.A.2
DelloRusso, C.3
Duan, D.4
Crawford, R.W.5
Phelps, S.F.6
-
2
-
-
79955158683
-
Systemic administration of PRO051 in Duchenne's muscular dystrophy
-
Goemans, NM, Tulinius, M, van den Akker, JT, Burm, BE, Ekhart, PF, Heuvelmans, N et al. (2011). Systemic administration of PRO051 in Duchenne's muscular dystrophy. N Engl J Med 364: 1513-1522.
-
(2011)
N Engl J Med
, vol.364
, pp. 1513-1522
-
-
Goemans, N.M.1
Tulinius, M.2
Van Den Akker, J.T.3
Burm, B.E.4
Ekhart, P.F.5
Heuvelmans, N.6
-
3
-
-
80051690306
-
Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: An open-label, phase 2, dose-escalation study
-
Cirak, S, Arechavala-Gomeza, V, Guglieri, M, Feng, L, Torelli, S, Anthony, K et al. (2011). Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: An open-label, phase 2, dose-escalation study. Lancet 378: 595-605.
-
(2011)
Lancet
, vol.378
, pp. 595-605
-
-
Cirak, S.1
Arechavala-Gomeza, V.2
Guglieri, M.3
Feng, L.4
Torelli, S.5
Anthony, K.6
-
4
-
-
0037364057
-
U7 snRNAs induce correction of mutated dystrophin pre-mRNA by exon skipping
-
Brun, C, Suter, D, Pauli, C, Dunant, P, Lochmüller, H, Burgunder, JM et al. (2003). U7 snRNAs induce correction of mutated dystrophin pre-mRNA by exon skipping. Cell Mol Life Sci 60: 557-566.
-
(2003)
Cell Mol Life Sci
, vol.60
, pp. 557-566
-
-
Brun, C.1
Suter, D.2
Pauli, C.3
Dunant, P.4
Lochmüller, H.5
Burgunder, J.M.6
-
5
-
-
10044240371
-
Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping
-
Goyenvalle, A, Vulin, A, Fougerousse, F, Leturcq, F, Kaplan, JC, Garcia, L et al. (2004). Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping. Science 306: 1796-1799.
-
(2004)
Science
, vol.306
, pp. 1796-1799
-
-
Goyenvalle, A.1
Vulin, A.2
Fougerousse, F.3
Leturcq, F.4
Kaplan, J.C.5
Garcia, L.6
-
6
-
-
33646930313
-
Chimeric adeno-associated virus/antisense U1 small nuclear RNA effectively rescues dystrophin synthesis and muscle function by local treatment of mdx mice
-
Denti, MA, Rosa, A, D'Antona, G, Sthandier, O, De Angelis, FG, Nicoletti, C et al. (2006). Chimeric adeno-associated virus/antisense U1 small nuclear RNA effectively rescues dystrophin synthesis and muscle function by local treatment of mdx mice. Hum Gene Ther 17: 565-574.
-
(2006)
Hum Gene Ther
, vol.17
, pp. 565-574
-
-
Denti, M.A.1
Rosa, A.2
D'Antona, G.3
Sthandier, O.4
De Angelis, F.G.5
Nicoletti, C.6
-
7
-
-
36749032678
-
Restoration of human dystrophin following transplantation of exon-skippingengineered DMD patient stem cells into dystrophic mice
-
Benchaouir, R, Meregalli, M, Farini, A, D'Antona, G, Belicchi, M, Goyenvalle, A et al. (2007). Restoration of human dystrophin following transplantation of exon-skippingengineered DMD patient stem cells into dystrophic mice. Cell Stem Cell 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
-
8
-
-
84861915467
-
Engineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophy
-
Goyenvalle, A, Wright, J, Babbs, A, Wilkins, V, Garcia, L and Davies, KE (2012). Engineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophy. Mol Ther 20: 1212-1221.
-
(2012)
Mol Ther
, vol.20
, pp. 1212-1221
-
-
Goyenvalle, A.1
Wright, J.2
Babbs, A.3
Wilkins, V.4
Garcia, L.5
Davies, K.E.6
-
9
-
-
84861131280
-
Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNAmediated exon skipping
-
Goyenvalle, A, Babbs, A, Wright, J, Wilkins, V, Powell, D, Garcia, L et al. (2012). Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNAmediated exon skipping. Hum Mol Genet 21: 2559-2571.
-
(2012)
Hum Mol Genet
, vol.21
, pp. 2559-2571
-
-
Goyenvalle, A.1
Babbs, A.2
Wright, J.3
Wilkins, V.4
Powell, D.5
Garcia, L.6
-
10
-
-
84857793829
-
Long-term restoration of cardiac dystrophin expression in golden retriever muscular dystrophy following rAAV6-mediated exon skipping
-
Bish, LT, Sleeper, MM, Forbes, SC, Wang, B, Reynolds, C, Singletary, GE et al. (2012). Long-term restoration of cardiac dystrophin expression in golden retriever muscular dystrophy following rAAV6-mediated exon skipping. Mol Ther 20: 580-589.
-
(2012)
Mol Ther
, vol.20
, pp. 580-589
-
-
Bish, L.T.1
Sleeper, M.M.2
Forbes, S.C.3
Wang, B.4
Reynolds, C.5
Singletary, G.E.6
-
11
-
-
84869086454
-
Muscle function recovery in golden retriever muscular dystrophy after AAV1-U7 exon skipping
-
Vulin, A, Barthélémy, I, Goyenvalle, A, Thibaud, JL, Beley, C, Griffith, G et al. (2012). Muscle function recovery in golden retriever muscular dystrophy after AAV1-U7 exon skipping. Mol Ther 20: 2120-2133.
-
(2012)
Mol Ther
, vol.20
, pp. 2120-2133
-
-
Vulin, A.1
Barthélémy, I.2
Goyenvalle, A.3
Thibaud, J.L.4
Beley, C.5
Griffith, G.6
-
12
-
-
13544262341
-
Long-term pharmacologically regulated expression of erythropoietin in primates following AAV-mediated gene transfer
-
Rivera, VM, Gao, GP, Grant, RL, Schnell, MA, Zoltick, PW, Rozamus, LW et al. (2005). Long-term pharmacologically regulated expression of erythropoietin in primates following AAV-mediated gene transfer. Blood 105: 1424-1430.
-
(2005)
Blood
, vol.105
, pp. 1424-1430
-
-
Rivera, V.M.1
Gao, G.P.2
Grant, R.L.3
Schnell, M.A.4
Zoltick, P.W.5
Rozamus, L.W.6
-
13
-
-
49149113894
-
Adeno-associated virus vector genomes persist as episomal chromatin in primate muscle
-
Penaud-Budloo, M, Le Guiner, C, Nowrouzi, A, Toromanoff, A, Chérel, Y, Chenuaud, P et al. (2008). Adeno-associated virus vector genomes persist as episomal chromatin in primate muscle. J Virol 82: 7875-7885.
-
(2008)
J Virol
, vol.82
, pp. 7875-7885
-
-
Penaud-Budloo, M.1
Le Guiner, C.2
Nowrouzi, A.3
Toromanoff, A.4
Chérel, Y.5
Chenuaud, P.6
-
14
-
-
39849091800
-
Transient immunomodulation allows repeated injections of AAV1 and correction of muscular dystrophy in multiple muscles
-
Lorain, S, Gross, DA, Goyenvalle, A, Danos, O, Davoust, J and Garcia, L (2008). Transient immunomodulation allows repeated injections of AAV1 and correction of muscular dystrophy in multiple muscles. Mol Ther 16: 541-547.
-
(2008)
Mol Ther
, vol.16
, pp. 541-547
-
-
Lorain, S.1
Gross, D.A.2
Goyenvalle, A.3
Danos, O.4
Davoust, J.5
Garcia, L.6
-
15
-
-
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. (1997). Utrophin-dystrophin-deficient mice as a model for Duchenne muscular dystrophy. Cell 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
-
16
-
-
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 and Sanes, JR (1997). Skeletal and cardiac myopathies in mice lacking utrophin and dystrophin: A model for Duchenne muscular dystrophy. Cell 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
-
17
-
-
0023935674
-
Regeneration of muscles after cardiotoxin injury. I. Cytological aspects
-
Couteaux, R, Mira, JC and d'Albis, A (1988). Regeneration of muscles after cardiotoxin injury. I. Cytological aspects. Biol Cell 62: 171-182.
-
(1988)
Biol Cell
, vol.62
, pp. 171-182
-
-
Couteaux, R.1
Mira, J.C.2
D'Albis, A.3
-
18
-
-
29244458622
-
Noninvasive monitoring of therapeutic gene transfer in animal models of muscular dystrophies
-
Bartoli, M, Poupiot, J, Goyenvalle, A, Perez, N, Garcia, L, Danos, O et al. (2006). Noninvasive monitoring of therapeutic gene transfer in animal models of muscular dystrophies. Gene Ther 13: 20-28.
-
(2006)
Gene Ther
, vol.13
, pp. 20-28
-
-
Bartoli, M.1
Poupiot, J.2
Goyenvalle, A.3
Perez, N.4
Garcia, L.5
Danos, O.6
-
19
-
-
77952011904
-
Combination of myostatin pathway interference and dystrophin rescue enhances tetanic and specific force in dystrophic mdx mice
-
Dumonceaux, J, Marie, S, Beley, C, Trollet, C, Vignaud, A, Ferry, A et al. (2010). Combination of myostatin pathway interference and dystrophin rescue enhances tetanic and specific force in dystrophic mdx mice. Mol Ther 18: 881-887.
-
(2010)
Mol Ther
, vol.18
, pp. 881-887
-
-
Dumonceaux, J.1
Marie, S.2
Beley, C.3
Trollet, C.4
Vignaud, A.5
Ferry, A.6
-
20
-
-
54949105845
-
Relative persistence of AAV serotype 1 vector genomes in dystrophic muscle
-
Pacak, CA, Conlon, T, Mah, CS and Byrne, BJ (2008). Relative persistence of AAV serotype 1 vector genomes in dystrophic muscle. Genet Vaccines Ther 6: 14.
-
(2008)
Genet Vaccines Ther
, vol.6
, pp. 14
-
-
Pacak, C.A.1
Conlon, T.2
Mah, C.S.3
Byrne, B.J.4
-
21
-
-
33745894322
-
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. (2006). rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice. Nat Med 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
-
22
-
-
0036537529
-
Adeno-associated virus vector gene transfer and sarcolemmal expression of a 144 kDa micro-dystrophin effectively restores the dystrophin-associated protein complex and inhibits myofibre degeneration in nude/mdx mice
-
Fabb, SA, Wells, DJ, Serpente, P and Dickson, G (2002). Adeno-associated virus vector gene transfer and sarcolemmal expression of a 144 kDa micro-dystrophin effectively restores the dystrophin-associated protein complex and inhibits myofibre degeneration in nude/mdx mice. Hum Mol Genet 11: 733-741.
-
(2002)
Hum Mol Genet
, vol.11
, pp. 733-741
-
-
Fabb, S.A.1
Wells, D.J.2
Serpente, P.3
Dickson, G.4
-
23
-
-
0034610364
-
Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model
-
Wang, B, Li, J and Xiao, X (2000). Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model. Proc Natl Acad Sci USA 97: 13714-13719.
-
(2000)
Proc Natl Acad Sci USA
, vol.97
, pp. 13714-13719
-
-
Wang, B.1
Li, J.2
Xiao, X.3
-
24
-
-
77957725001
-
Dystrophin immunity in Duchenne's muscular dystrophy
-
Mendell, JR, Campbell, K, Rodino-Klapac, L, Sahenk, Z, Shilling, C, Lewis, S et al. (2010). Dystrophin immunity in Duchenne's muscular dystrophy. N Engl J Med 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
-
25
-
-
84864558640
-
Successful regional delivery and long-term expression of a dystrophin gene in canine muscular dystrophy: A preclinical model for human therapies
-
Wang, Z, Storb, R, Halbert, CL, Banks, GB, Butts, TM, Finn, EE et al. (2012). Successful regional delivery and long-term expression of a dystrophin gene in canine muscular dystrophy: A preclinical model for human therapies. Mol Ther 20: 1501-1507.
-
(2012)
Mol Ther
, vol.20
, pp. 1501-1507
-
-
Wang, Z.1
Storb, R.2
Halbert, C.L.3
Banks, G.B.4
Butts, T.M.5
Finn, E.E.6
-
26
-
-
77953134497
-
Preclinical PK and PD studies on 2'-O-methyl-phosphorothioate RNA antisense oligonucleotides in the mdx mouse model
-
Heemskerk, H, de Winter, C, van Kuik, P, Heuvelmans, N, Sabatelli, P, Rimessi, P et al. (2010). Preclinical PK and PD studies on 2'-O-methyl- phosphorothioate RNA antisense oligonucleotides in the mdx mouse model. Mol Ther 18: 1210-1217.
-
(2010)
Mol Ther
, vol.18
, pp. 1210-1217
-
-
Heemskerk, H.1
De Winter, C.2
Van Kuik, P.3
Heuvelmans, N.4
Sabatelli, P.5
Rimessi, P.6
-
27
-
-
37549049805
-
Skipping toward personalized molecular medicine
-
Hoffman, EP (2007). Skipping toward personalized molecular medicine. N Engl J Med 357: 2719-2722.
-
(2007)
N Engl J Med
, vol.357
, pp. 2719-2722
-
-
Hoffman, E.P.1
-
28
-
-
36248985708
-
Dystrophin levels as low as 30% are sufficient to avoid muscular dystrophy in the human
-
Neri, M, Torelli, S, Brown, S, Ugo, I, Sabatelli, P, Merlini, L et al. (2007). Dystrophin levels as low as 30% are sufficient to avoid muscular dystrophy in the human. Neuromuscul Disord 17: 913-918.
-
(2007)
Neuromuscul Disord
, vol.17
, pp. 913-918
-
-
Neri, M.1
Torelli, S.2
Brown, S.3
Ugo, I.4
Sabatelli, P.5
Merlini, L.6
-
29
-
-
84870215284
-
A marginal level of dystrophin partially ameliorates hindlimb muscle passive mechanical properties in dystrophin-null mice
-
Hakim, CH and Duan, D (2012). A marginal level of dystrophin partially ameliorates hindlimb muscle passive mechanical properties in dystrophin-null mice. Muscle Nerve 46: 948-950.
-
(2012)
Muscle Nerve
, vol.46
, pp. 948-950
-
-
Hakim, C.H.1
Duan, D.2
|