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Journal Abstract Search

286 related items for PubMed ID: 20154340

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  • 4. Efficient bypass of mutations in dysferlin deficient patient cells by antisense-induced exon skipping.
    Wein N, Avril A, Bartoli M, Beley C, Chaouch S, Laforêt P, Behin A, Butler-Browne G, Mouly V, Krahn M, Garcia L, Lévy N.
    Hum Mutat; 2010 Feb; 31(2):136-42. PubMed ID: 19953532
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  • 6. rAAV vector-mediated sarcogylcan gene transfer in a hamster model for limb girdle muscular dystrophy.
    Li J, Dressman D, Tsao YP, Sakamoto A, Hoffman EP, Xiao X.
    Gene Ther; 1999 Jan; 6(1):74-82. PubMed ID: 10341878
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  • 7. A naturally occurring human minidysferlin protein repairs sarcolemmal lesions in a mouse model of dysferlinopathy.
    Krahn M, Wein N, Bartoli M, Lostal W, Courrier S, Bourg-Alibert N, Nguyen K, Vial C, Streichenberger N, Labelle V, DePetris D, Pécheux C, Leturcq F, Cau P, Richard I, Lévy N.
    Sci Transl Med; 2010 Sep 22; 2(50):50ra69. PubMed ID: 20861509
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  • 8. Disruption of muscle membrane and phenotype divergence in two novel mouse models of dysferlin deficiency.
    Ho M, Post CM, Donahue LR, Lidov HG, Bronson RT, Goolsby H, Watkins SC, Cox GA, Brown RH.
    Hum Mol Genet; 2004 Sep 15; 13(18):1999-2010. PubMed ID: 15254015
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  • 9. Mutation finding in patients with dysferlin deficiency and role of the dysferlin interacting proteins annexin A1 and A2 in muscular dystrophies.
    Cagliani R, Magri F, Toscano A, Merlini L, Fortunato F, Lamperti C, Rodolico C, Prelle A, Sironi M, Aguennouz M, Ciscato P, Uncini A, Moggio M, Bresolin N, Comi GP.
    Hum Mutat; 2005 Sep 15; 26(3):283. PubMed ID: 16100712
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  • 11. Adeno-associated virus vector-mediated gene transfer into dystrophin-deficient skeletal muscles evokes enhanced immune response against the transgene product.
    Yuasa K, Sakamoto M, Miyagoe-Suzuki Y, Tanouchi A, Yamamoto H, Li J, Chamberlain JS, Xiao X, Takeda S.
    Gene Ther; 2002 Dec 15; 9(23):1576-88. PubMed ID: 12424610
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  • 13. Lariat branch point mutation in the dysferlin gene with mild limb-girdle muscular dystrophy.
    Sinnreich M, Therrien C, Karpati G.
    Neurology; 2006 Apr 11; 66(7):1114-6. PubMed ID: 16606933
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  • 19. Homologous recombination mediates functional recovery of dysferlin deficiency following AAV5 gene transfer.
    Grose WE, Clark KR, Griffin D, Malik V, Shontz KM, Montgomery CL, Lewis S, Brown RH, Janssen PM, Mendell JR, Rodino-Klapac LR.
    PLoS One; 2012 Apr 11; 7(6):e39233. PubMed ID: 22720081
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  • 20. A canine minidystrophin is functional and therapeutic in mdx mice.
    Wang B, Li J, Qiao C, Chen C, Hu P, Zhu X, Zhou L, Bogan J, Kornegay J, Xiao X.
    Gene Ther; 2008 Aug 11; 15(15):1099-106. PubMed ID: 18432277
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