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Journal Abstract Search

237 related items for PubMed ID: 27401142

  • 1. Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal Muscular Atrophy.
    Osman EY, Washington CW, Kaifer KA, Mazzasette C, Patitucci TN, Florea KM, Simon ME, Ko CP, Ebert AD, Lorson CL.
    Mol Ther; 2016 Sep; 24(9):1592-601. PubMed ID: 27401142
    [Abstract] [Full Text] [Related]

  • 2. Morpholino antisense oligonucleotides targeting intronic repressor Element1 improve phenotype in SMA mouse models.
    Osman EY, Miller MR, Robbins KL, Lombardi AM, Atkinson AK, Brehm AJ, Lorson CL.
    Hum Mol Genet; 2014 Sep 15; 23(18):4832-45. PubMed ID: 24781211
    [Abstract] [Full Text] [Related]

  • 3. Improved antisense oligonucleotide design to suppress aberrant SMN2 gene transcript processing: towards a treatment for spinal muscular atrophy.
    Mitrpant C, Porensky P, Zhou H, Price L, Muntoni F, Fletcher S, Wilton SD, Burghes AH.
    PLoS One; 2013 Sep 15; 8(4):e62114. PubMed ID: 23630626
    [Abstract] [Full Text] [Related]

  • 4. Bifunctional RNAs targeting the intronic splicing silencer N1 increase SMN levels and reduce disease severity in an animal model of spinal muscular atrophy.
    Osman EY, Yen PF, Lorson CL.
    Mol Ther; 2012 Jan 15; 20(1):119-26. PubMed ID: 22031236
    [Abstract] [Full Text] [Related]

  • 5. Optimization of SMN trans-splicing through the analysis of SMN introns.
    Shababi M, Lorson CL.
    J Mol Neurosci; 2012 Mar 15; 46(3):459-69. PubMed ID: 21826391
    [Abstract] [Full Text] [Related]

  • 6. 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]

  • 7. Combination of valproic acid and morpholino splice-switching oligonucleotide produces improved outcomes in spinal muscular atrophy patient-derived fibroblasts.
    Farrelly-Rosch A, Lau CL, Patil N, Turner BJ, Shabanpoor F.
    Neurochem Int; 2017 Sep 01; 108():213-221. PubMed ID: 28389270
    [Abstract] [Full Text] [Related]

  • 8. A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse.
    Porensky PN, Mitrpant C, McGovern VL, Bevan AK, Foust KD, Kaspar BK, Wilton SD, Burghes AH.
    Hum Mol Genet; 2012 Apr 01; 21(7):1625-38. PubMed ID: 22186025
    [Abstract] [Full Text] [Related]

  • 9. 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]

  • 10. 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]

  • 11. Development and characterization of an SMN2-based intermediate mouse model of Spinal Muscular Atrophy.
    Cobb MS, Rose FF, Rindt H, Glascock JJ, Shababi M, Miller MR, Osman EY, Yen PF, Garcia ML, Martin BR, Wetz MJ, Mazzasette C, Feng Z, Ko CP, Lorson CL.
    Hum Mol Genet; 2013 May 01; 22(9):1843-55. PubMed ID: 23390132
    [Abstract] [Full Text] [Related]

  • 12. Recent Advances and Clinical Applications of Exon Inclusion for Spinal Muscular Atrophy.
    Son HW, Yokota T.
    Methods Mol Biol; 2018 May 01; 1828():57-68. PubMed ID: 30171534
    [Abstract] [Full Text] [Related]

  • 13. 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 May 01; 11(4):e0154390. PubMed ID: 27111068
    [Abstract] [Full Text] [Related]

  • 14. Splicing of a critical exon of human Survival Motor Neuron is regulated by a unique silencer element located in the last intron.
    Singh NK, Singh NN, Androphy EJ, Singh RN.
    Mol Cell Biol; 2006 Feb 01; 26(4):1333-46. PubMed ID: 16449646
    [Abstract] [Full Text] [Related]

  • 15. Delivery of bifunctional RNAs that target an intronic repressor and increase SMN levels in an animal model of spinal muscular atrophy.
    Baughan TD, Dickson A, Osman EY, Lorson CL.
    Hum Mol Genet; 2009 May 01; 18(9):1600-11. PubMed ID: 19228773
    [Abstract] [Full Text] [Related]

  • 16. A multi-exon-skipping detection assay reveals surprising diversity of splice isoforms of spinal muscular atrophy genes.
    Singh NN, Seo J, Rahn SJ, Singh RN.
    PLoS One; 2012 May 01; 7(11):e49595. PubMed ID: 23185376
    [Abstract] [Full Text] [Related]

  • 17. Investigation of New Morpholino Oligomers to Increase Survival Motor Neuron Protein Levels in Spinal Muscular Atrophy.
    Ramirez A, Crisafulli SG, Rizzuti M, Bresolin N, Comi GP, Corti S, Nizzardo M.
    Int J Mol Sci; 2018 Jan 06; 19(1):. PubMed ID: 29316633
    [Abstract] [Full Text] [Related]

  • 18. NCALD Antisense Oligonucleotide Therapy in Addition to Nusinersen further Ameliorates Spinal Muscular Atrophy in Mice.
    Torres-Benito L, Schneider S, Rombo R, Ling KK, Grysko V, Upadhyay A, Kononenko NL, Rigo F, Bennett CF, Wirth B.
    Am J Hum Genet; 2019 Jul 03; 105(1):221-230. PubMed ID: 31230718
    [Abstract] [Full Text] [Related]

  • 19. Spinal muscular atrophy phenotype is ameliorated in human motor neurons by SMN increase via different novel RNA therapeutic approaches.
    Nizzardo M, Simone C, Dametti S, Salani S, Ulzi G, Pagliarani S, Rizzo F, Frattini E, Pagani F, Bresolin N, Comi G, Corti S.
    Sci Rep; 2015 Jun 30; 5():11746. PubMed ID: 26123042
    [Abstract] [Full Text] [Related]

  • 20. Alternative splicing in spinal muscular atrophy underscores the role of an intron definition model.
    Singh NN, Singh RN.
    RNA Biol; 2011 Jun 30; 8(4):600-6. PubMed ID: 21654213
    [Abstract] [Full Text] [Related]

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