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Journal Abstract Search


1376 related items for PubMed ID: 21979052

  • 1. Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model.
    Hua Y, Sahashi K, Rigo F, Hung G, Horev G, Bennett CF, Krainer AR.
    Nature; 2011 Oct 05; 478(7367):123-6. PubMed ID: 21979052
    [Abstract] [Full Text] [Related]

  • 2. Normalization of Patient-Identified Plasma Biomarkers in SMNΔ7 Mice following Postnatal SMN Restoration.
    Arnold WD, Duque S, Iyer CC, Zaworski P, McGovern VL, Taylor SJ, von Herrmann KM, Kobayashi DT, Chen KS, Kolb SJ, Paushkin SV, Burghes AH.
    PLoS One; 2016 Oct 05; 11(12):e0167077. PubMed ID: 27907033
    [Abstract] [Full Text] [Related]

  • 3. Pathological impact of SMN2 mis-splicing in adult SMA mice.
    Sahashi K, Ling KK, Hua Y, Wilkinson JE, Nomakuchi T, Rigo F, Hung G, Xu D, Jiang YP, Lin RZ, Ko CP, Bennett CF, Krainer AR.
    EMBO Mol Med; 2013 Oct 05; 5(10):1586-601. PubMed ID: 24014320
    [Abstract] [Full Text] [Related]

  • 4. Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model.
    Hua Y, Sahashi K, Hung G, Rigo F, Passini MA, Bennett CF, Krainer AR.
    Genes Dev; 2010 Aug 01; 24(15):1634-44. PubMed ID: 20624852
    [Abstract] [Full Text] [Related]

  • 5. Nusinersen ameliorates motor function and prevents motoneuron Cajal body disassembly and abnormal poly(A) RNA distribution in a SMA mouse model.
    Berciano MT, Puente-Bedia A, Medina-Samamé A, Rodríguez-Rey JC, Calderó J, Lafarga M, Tapia O.
    Sci Rep; 2020 Jul 01; 10(1):10738. PubMed ID: 32612161
    [Abstract] [Full Text] [Related]

  • 6. Oligonucleotide-mediated survival of motor neuron protein expression in CNS improves phenotype in a mouse model of spinal muscular atrophy.
    Williams JH, Schray RC, Patterson CA, Ayitey SO, Tallent MK, Lutz GJ.
    J Neurosci; 2009 Jun 17; 29(24):7633-8. PubMed ID: 19535574
    [Abstract] [Full Text] [Related]

  • 7. Spinal muscular atrophy: antisense oligonucleotide therapy opens the door to an integrated therapeutic landscape.
    Wood MJA, Talbot K, Bowerman M.
    Hum Mol Genet; 2017 Oct 01; 26(R2):R151-R159. PubMed ID: 28977438
    [Abstract] [Full Text] [Related]

  • 8. RNA-sequencing of a mouse-model of spinal muscular atrophy reveals tissue-wide changes in splicing of U12-dependent introns.
    Doktor TK, Hua Y, Andersen HS, Brøner S, Liu YH, Wieckowska A, Dembic M, Bruun GH, Krainer AR, Andresen BS.
    Nucleic Acids Res; 2017 Jan 09; 45(1):395-416. PubMed ID: 27557711
    [Abstract] [Full Text] [Related]

  • 9. Histopathological Defects in Intestine in Severe Spinal Muscular Atrophy Mice Are Improved by Systemic Antisense Oligonucleotide Treatment.
    Sintusek P, Catapano F, Angkathunkayul N, Marrosu E, Parson SH, Morgan JE, Muntoni F, Zhou H.
    PLoS One; 2016 Jan 09; 11(5):e0155032. PubMed ID: 27163330
    [Abstract] [Full Text] [Related]

  • 10. Oxidative Stress Triggers Body-Wide Skipping of Multiple Exons of the Spinal Muscular Atrophy Gene.
    Seo J, Singh NN, Ottesen EW, Sivanesan S, Shishimorova M, Singh RN.
    PLoS One; 2016 Jan 09; 11(4):e0154390. PubMed ID: 27111068
    [Abstract] [Full Text] [Related]

  • 11. Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy.
    Passini MA, Bu J, Richards AM, Kinnecom C, Sardi SP, Stanek LM, Hua Y, Rigo F, Matson J, Hung G, Kaye EM, Shihabuddin LS, Krainer AR, Bennett CF, Cheng SH.
    Sci Transl Med; 2011 Mar 02; 3(72):72ra18. PubMed ID: 21368223
    [Abstract] [Full Text] [Related]

  • 12. ZPR1 prevents R-loop accumulation, upregulates SMN2 expression and rescues spinal muscular atrophy.
    Kannan A, Jiang X, He L, Ahmad S, Gangwani L.
    Brain; 2020 Jan 01; 143(1):69-93. PubMed ID: 31828288
    [Abstract] [Full Text] [Related]

  • 13. A SMN missense mutation complements SMN2 restoring snRNPs and rescuing SMA mice.
    Workman E, Saieva L, Carrel TL, Crawford TO, Liu D, Lutz C, Beattie CE, Pellizzoni L, Burghes AH.
    Hum Mol Genet; 2009 Jun 15; 18(12):2215-29. PubMed ID: 19329542
    [Abstract] [Full Text] [Related]

  • 14. Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy.
    Zhao X, Feng Z, Ling KK, Mollin A, Sheedy J, Yeh S, Petruska J, Narasimhan J, Dakka A, Welch EM, Karp G, Chen KS, Metzger F, Ratni H, Lotti F, Tisdale S, Naryshkin NA, Pellizzoni L, Paushkin S, Ko CP, Weetall M.
    Hum Mol Genet; 2016 May 15; 25(10):1885-1899. PubMed ID: 26931466
    [Abstract] [Full Text] [Related]

  • 15. The zinc finger protein ZPR1 is a potential modifier of spinal muscular atrophy.
    Ahmad S, Wang Y, Shaik GM, Burghes AH, Gangwani L.
    Hum Mol Genet; 2012 Jun 15; 21(12):2745-58. PubMed ID: 22422766
    [Abstract] [Full Text] [Related]

  • 16. Temporal and tissue-specific variability of SMN protein levels in mouse models of spinal muscular atrophy.
    Groen EJN, Perenthaler E, Courtney NL, Jordan CY, Shorrock HK, van der Hoorn D, Huang YT, Murray LM, Viero G, Gillingwater TH.
    Hum Mol Genet; 2018 Aug 15; 27(16):2851-2862. PubMed ID: 29790918
    [Abstract] [Full Text] [Related]

  • 17. SMN deficiency alters Nrxn2 expression and splicing in zebrafish and mouse models of spinal muscular atrophy.
    See K, Yadav P, Giegerich M, Cheong PS, Graf M, Vyas H, Lee SG, Mathavan S, Fischer U, Sendtner M, Winkler C.
    Hum Mol Genet; 2014 Apr 01; 23(7):1754-70. PubMed ID: 24218366
    [Abstract] [Full Text] [Related]

  • 18. Motor neuron cell-nonautonomous rescue of spinal muscular atrophy phenotypes in mild and severe transgenic mouse models.
    Hua Y, Liu YH, Sahashi K, Rigo F, Bennett CF, Krainer AR.
    Genes Dev; 2015 Feb 01; 29(3):288-97. PubMed ID: 25583329
    [Abstract] [Full Text] [Related]

  • 19. Effect of combined systemic and local morpholino treatment on the spinal muscular atrophy Δ7 mouse model phenotype.
    Nizzardo M, Simone C, Salani S, Ruepp MD, Rizzo F, Ruggieri M, Zanetta C, Brajkovic S, Moulton HM, Müehlemann O, Bresolin N, Comi GP, Corti S.
    Clin Ther; 2014 Mar 01; 36(3):340-56.e5. PubMed ID: 24636820
    [Abstract] [Full Text] [Related]

  • 20. The Antisense Transcript SMN-AS1 Regulates SMN Expression and Is a Novel Therapeutic Target for Spinal Muscular Atrophy.
    d'Ydewalle C, Ramos DM, Pyles NJ, Ng SY, Gorz M, Pilato CM, Ling K, Kong L, Ward AJ, Rubin LL, Rigo F, Bennett CF, Sumner CJ.
    Neuron; 2017 Jan 04; 93(1):66-79. PubMed ID: 28017471
    [Abstract] [Full Text] [Related]


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