Emery AE: Population frequencies of inherited neuromuscular diseases--a world survey. Neuromuscul Disord. 1991, 1 (1): 19-29. 10.1016/0960-8966(91)90039-U.
Article
CAS
PubMed
Google Scholar
Mendell JR, Moxley RT, Griggs RC, Brooke MH, Fenichel GM, Miller JP, King W, Signore L, Pandya S, Florence J: Randomized, double-blind six-month trial of prednisone in Duchenne's muscular dystrophy. N Engl J Med. 1989, 320 (24): 1592-1597.
Article
CAS
PubMed
Google Scholar
Aartsma-Rus A, Kaman WE, Weij R, den Dunnen JT, van Ommen GJ, van Deutekom JC: Exploring the frontiers of therapeutic exon skipping for Duchenne muscular dystrophy by double targeting within one or multiple exons. Mol Ther. 2006, 14 (3): 401-407. 10.1016/j.ymthe.2006.02.022.
Article
CAS
PubMed
Google Scholar
McClorey G, Moulton HM, Iversen PL, Fletcher S, Wilton SD: Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD. Gene Ther. 2006, 13 (19): 1373-1381. 10.1038/sj.gt.3302800.
Article
CAS
PubMed
Google Scholar
Barton-Davis ER, Cordier L, Shoturma DI, Leland SE, Sweeney HL: Aminoglycoside antibiotics restore dystrophin function to skeletal muscles of mdx mice. J Clin Invest. 1999, 104 (4): 375-381.
Article
PubMed Central
CAS
PubMed
Google Scholar
Hamed SA: Drug evaluation: PTC-124--a potential treatment of cystic fibrosis and Duchenne muscular dystrophy. IDrugs. 2006, 9 (11): 783-789.
CAS
PubMed
Google Scholar
Tinsley J, Deconinck N, Fisher R, Kahn D, Phelps S, Gillis JM, Davies K: Expression of full-length utrophin prevents muscular dystrophy in mdx mice. Nat Med. 1998, 4 (12): 1441-1444. 10.1038/4033.
Article
CAS
PubMed
Google Scholar
Bogdanovich S, Krag TO, Barton ER, Morris LD, Whittemore LA, Ahima RS, Khurana TS: Functional improvement of dystrophic muscle by myostatin blockade. Nature. 2002, 420 (6914): 418-421. 10.1038/nature01154.
Article
CAS
PubMed
Google Scholar
Gregorevic P, Allen JM, Minami E, Blankinship MJ, Haraguchi M, Meuse L, Finn E, Adams ME, Froehner SC, Murry CE, Chamberlain JS: rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice. Nat Med. 2006, 12 (7): 787-789. 10.1038/nm1439.
Article
PubMed Central
CAS
PubMed
Google Scholar
Gregorevic P, Blankinship MJ, Allen JM, Crawford RW, Meuse L, Miller DG, Russell DW, Chamberlain JS: Systemic delivery of genes to striated muscles using adeno-associated viral vectors. Nat Med. 2004, 10 (8): 828-834. 10.1038/nm1085.
Article
PubMed Central
CAS
PubMed
Google Scholar
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 (2): 245-256. 10.1016/j.ymthe.2004.09.013.
Article
PubMed Central
CAS
PubMed
Google Scholar
Yoshimura M, Sakamoto M, Ikemoto M, Mochizuki Y, Yuasa K, Miyagoe-Suzuki Y, Takeda S: AAV vector-mediated microdystrophin expression in a relatively small percentage of mdx myofibers improved the mdx phenotype. Mol Ther. 2004, 10 (5): 821-828. 10.1016/j.ymthe.2004.07.025.
Article
CAS
PubMed
Google Scholar
Sampaolesi M, Blot S, D'Antona G, Granger N, Tonlorenzi R, Innocenzi A, Mognol P, Thibaud JL, Galvez BG, Barthelemy I, Perani L, Mantero S, Guttinger M, Pansarasa O, Rinaldi C, Cusella De Angelis MG, Torrente Y, Bordignon C, Bottinelli R, Cossu G: Mesoangioblast stem cells ameliorate muscle function in dystrophic dogs. Nature. 2006, 444 (7119): 574-579. 10.1038/nature05282.
Article
CAS
PubMed
Google Scholar
Cerletti M, Negri T, Cozzi F, Colpo R, Andreetta F, Croci D, Davies KE, Cornelio F, Pozza O, Karpati G, Gilbert R, Mora M: Dystrophic phenotype of canine X-linked muscular dystrophy is mitigated by adenovirus-mediated utrophin gene transfer. Gene Ther. 2003, 10 (9): 750-757. 10.1038/sj.gt.3301941.
Article
CAS
PubMed
Google Scholar
Wang Z, Zhu T, Qiao C, Zhou L, Wang B, Zhang J, Chen C, Li J, Xiao X: Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart. Nat Biotechnol. 2005, 23 (3): 321-328. 10.1038/nbt1073.
Article
CAS
PubMed
Google Scholar
Xiao W, Chirmule N, Berta SC, McCullough B, Gao G, Wilson JM: Gene therapy vectors based on adeno-associated virus type 1. J Virol. 1999, 73 (5): 3994-4003.
PubMed Central
CAS
PubMed
Google Scholar
Schnepp BC, Clark KR, Klemanski DL, Pacak CA, Johnson PR: Genetic fate of recombinant adeno-associated virus vector genomes in muscle. J Virol. 2003, 77 (6): 3495-3504. 10.1128/JVI.77.6.3495-3504.2003.
Article
PubMed Central
CAS
PubMed
Google Scholar
Goyenvalle A, Vulin A, Fougerousse F, Leturcq F, Kaplan JC, Garcia L, Danos O: Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping. Science. 2004, 306 (5702): 1796-1799. 10.1126/science.1104297.
Article
CAS
PubMed
Google Scholar
Wang Z, Kuhr CS, Allen JM, Blankinship M, Gregorevic P, Chamberlain JS, Tapscott SJ, Storb R: Sustained AAV-mediated dystrophin expression in a canine model of Duchenne muscular dystrophy with a brief course of immunosuppression. Mol Ther. 2007, 15 (6): 1160-1166.
CAS
PubMed
Google Scholar
Harper SQ, Hauser MA, DelloRusso C, Duan D, Crawford RW, Phelps SF, Harper HA, Robinson AS, Engelhardt JF, Brooks SV, Chamberlain JS: Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy. Nat Med. 2002, 8 (3): 253-261. 10.1038/nm0302-253.
Article
CAS
PubMed
Google Scholar
Rabinowitz JE, Rolling F, Li C, Conrath H, Xiao W, Xiao X, Samulski RJ: Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity. J Virol. 2002, 76 (2): 791-801. 10.1128/JVI.76.2.791-801.2002.
Article
PubMed Central
CAS
PubMed
Google Scholar
Clark KR, Liu X, McGrath JP, Johnson PR: Highly purified recombinant adeno-associated virus vectors are biologically active and free of detectable helper and wild-type viruses. Hum Gene Ther. 1999, 10 (6): 1031-1039. 10.1089/10430349950018427.
Article
CAS
PubMed
Google Scholar
Palmiter RD, Sandgren EP, Avarbock MR, Allen DD, Brinster RL: Heterologous introns can enhance expression of transgenes in mice. Proc Natl Acad Sci U S A. 1991, 88 (2): 478-482. 10.1073/pnas.88.2.478.
Article
PubMed Central
CAS
PubMed
Google Scholar
Fougerousse F, Bartoli M, Poupiot J, Arandel L, Durand M, Guerchet N, Gicquel E, Danos O, Richard I: Phenotypic Correction of alpha-Sarcoglycan Deficiency by Intra-arterial Injection of a Muscle-specific Serotype 1 rAAV Vector. Mol Ther. 2007, 15 (1): 53-61. 10.1038/sj.mt.6300022.
Article
CAS
PubMed
Google Scholar
Gonin P, Arandel L, Van Wittenberghe L, Marais T, Perez N, Danos O: Femoral intra-arterial injection: a tool to deliver and assess recombinant AAV constructs in rodents whole hind limb. J Gene Med. 2005, 7 (6): 782-791. 10.1002/jgm.716.
Article
CAS
PubMed
Google Scholar
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 U S A. 2000, 97 (25): 13714-13719. 10.1073/pnas.240335297.
Article
PubMed Central
CAS
PubMed
Google Scholar
Hodges BL, Taylor KM, Chu Q, Scull SE, Serriello RG, Anderson SC, Wang F, Scheule RK: Local delivery of a viral vector mitigates neutralization by antiviral antibodies and results in efficient transduction of rabbit liver. Mol Ther. 2005, 12 (6): 1043-1051. 10.1016/j.ymthe.2005.06.475.
Article
CAS
PubMed
Google Scholar
Manno CS, Pierce GF, Arruda VR, Glader B, Ragni M, Rasko JJ, Ozelo MC, Hoots K, Blatt P, Konkle B, Dake M, Kaye R, Razavi M, Zajko A, Zehnder J, Rustagi PK, Nakai H, Chew A, Leonard D, Wright JF, Lessard RR, Sommer JM, Tigges M, Sabatino D, Luk A, Jiang H, Mingozzi F, Couto L, Ertl HC, High KA, Kay MA: Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med. 2006, 12 (3): 342-347. 10.1038/nm1358.
Article
CAS
PubMed
Google Scholar
Foster K, Foster H, Dickson JG: Gene therapy progress and prospects: Duchenne muscular dystrophy. Gene Ther. 2006, 13 (24): 1677-1685. 10.1038/sj.gt.3302877.
Article
CAS
PubMed
Google Scholar
Chirmule N, Propert K, Magosin S, Qian Y, Qian R, Wilson J: Immune responses to adenovirus and adeno-associated virus in humans. Gene Ther. 1999, 6 (9): 1574-1583. 10.1038/sj.gt.3300994.
Article
CAS
PubMed
Google Scholar