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Journal Abstract Search

252 related items for PubMed ID: 36995605

  • 21. In vitro evaluation of novel antisense oligonucleotides is predictive of in vivo exon skipping activity for Duchenne muscular dystrophy.
    Wang Q, Yin H, Camelliti P, Betts C, Moulton H, Lee H, Saleh AF, Gait MJ, Wood MJ.
    J Gene Med; 2010 Apr; 12(4):354-64. PubMed ID: 20235089
    [Abstract] [Full Text] [Related]

  • 22. Nonclinical Exon Skipping Studies with 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides in mdx and mdx-utrn-/- Mice Inspired by Clinical Trial Results.
    van Putten M, Tanganyika-de Winter C, Bosgra S, Aartsma-Rus A.
    Nucleic Acid Ther; 2019 Apr; 29(2):92-103. PubMed ID: 30672725
    [Abstract] [Full Text] [Related]

  • 23. Tips to Design Effective Splice-Switching Antisense Oligonucleotides for Exon Skipping and Exon Inclusion.
    Maruyama R, Yokota T.
    Methods Mol Biol; 2018 Apr; 1828():79-90. PubMed ID: 30171536
    [Abstract] [Full Text] [Related]

  • 24. Optimizing antisense oligonucleotides using phosphorodiamidate morpholino oligomers.
    Popplewell LJ, Malerba A, Dickson G.
    Methods Mol Biol; 2012 Apr; 867():143-67. PubMed ID: 22454060
    [Abstract] [Full Text] [Related]

  • 25. Pharmacology and toxicology of eteplirsen and SRP-5051 for DMD exon 51 skipping: an update.
    Sheikh O, Yokota T.
    Arch Toxicol; 2022 Jan; 96(1):1-9. PubMed ID: 34797383
    [Abstract] [Full Text] [Related]

  • 26. Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD.
    McClorey G, Moulton HM, Iversen PL, Fletcher S, Wilton SD.
    Gene Ther; 2006 Oct; 13(19):1373-81. PubMed ID: 16724091
    [Abstract] [Full Text] [Related]

  • 27. Skipping multiple exons of dystrophin transcripts using cocktail antisense oligonucleotides.
    Echigoya Y, Yokota T.
    Nucleic Acid Ther; 2014 Feb; 24(1):57-68. PubMed ID: 24380394
    [Abstract] [Full Text] [Related]

  • 28. Bioinformatic and functional optimization of antisense phosphorodiamidate morpholino oligomers (PMOs) for therapeutic modulation of RNA splicing in muscle.
    Popplewell LJ, Graham IR, Malerba A, Dickson G.
    Methods Mol Biol; 2011 Feb; 709():153-78. PubMed ID: 21194027
    [Abstract] [Full Text] [Related]

  • 29. Systemic administration of the antisense oligonucleotide NS-065/NCNP-01 for skipping of exon 53 in patients with Duchenne muscular dystrophy.
    Komaki H, Nagata T, Saito T, Masuda S, Takeshita E, Sasaki M, Tachimori H, Nakamura H, Aoki Y, Takeda S.
    Sci Transl Med; 2018 Apr 18; 10(437):. PubMed ID: 29669851
    [Abstract] [Full Text] [Related]

  • 30. In vivo comparison of 2'-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping.
    Heemskerk HA, de Winter CL, de Kimpe SJ, van Kuik-Romeijn P, Heuvelmans N, Platenburg GJ, van Ommen GJ, van Deutekom JC, Aartsma-Rus A.
    J Gene Med; 2009 Mar 18; 11(3):257-66. PubMed ID: 19140108
    [Abstract] [Full Text] [Related]

  • 31. Antisense PMO cocktails effectively skip dystrophin exons 45-55 in myotubes transdifferentiated from DMD patient fibroblasts.
    Lee J, Echigoya Y, Duddy W, Saito T, Aoki Y, Takeda S, Yokota T.
    PLoS One; 2018 Mar 18; 13(5):e0197084. PubMed ID: 29771942
    [Abstract] [Full Text] [Related]

  • 32. Targeted exon skipping in transgenic hDMD mice: A model for direct preclinical screening of human-specific antisense oligonucleotides.
    Bremmer-Bout M, Aartsma-Rus A, de Meijer EJ, Kaman WE, Janson AA, Vossen RH, van Ommen GJ, den Dunnen JT, van Deutekom JC.
    Mol Ther; 2004 Aug 18; 10(2):232-40. PubMed ID: 15294170
    [Abstract] [Full Text] [Related]

  • 33. Comparative analysis of antisense oligonucleotide sequences targeting exon 53 of the human DMD gene: Implications for future clinical trials.
    Popplewell LJ, Adkin C, Arechavala-Gomeza V, Aartsma-Rus A, de Winter CL, Wilton SD, Morgan JE, Muntoni F, Graham IR, Dickson G.
    Neuromuscul Disord; 2010 Feb 18; 20(2):102-10. PubMed ID: 20079639
    [Abstract] [Full Text] [Related]

  • 34. Dosing regimen has a significant impact on the efficiency of morpholino oligomer-induced exon skipping in mdx mice.
    Malerba A, Thorogood FC, Dickson G, Graham IR.
    Hum Gene Ther; 2009 Sep 18; 20(9):955-65. PubMed ID: 19469709
    [Abstract] [Full Text] [Related]

  • 35. Eteplirsen in the treatment of Duchenne muscular dystrophy.
    Lim KR, Maruyama R, Yokota T.
    Drug Des Devel Ther; 2017 Sep 18; 11():533-545. PubMed ID: 28280301
    [Abstract] [Full Text] [Related]

  • 36. Evaluation of Exon Skipping and Dystrophin Restoration in In Vitro Models of Duchenne Muscular Dystrophy.
    López-Martínez A, Soblechero-Martín P, Arechavala-Gomeza V.
    Methods Mol Biol; 2022 Sep 18; 2434():217-233. PubMed ID: 35213020
    [Abstract] [Full Text] [Related]

  • 37. [Exon skipping therapy for Duchenne muscular dystrophy by using antisense Morpholino].
    Takeda S.
    Rinsho Shinkeigaku; 2009 Nov 18; 49(11):856-8. PubMed ID: 20030230
    [Abstract] [Full Text] [Related]

  • 38. Systemic Injection of Peptide-PMOs into Humanized DMD Mice and Evaluation by RT-PCR and ELISA.
    Melo D, Maruyama R, Yokota T.
    Methods Mol Biol; 2018 Nov 18; 1828():263-273. PubMed ID: 30171547
    [Abstract] [Full Text] [Related]

  • 39. Extensive and prolonged restoration of dystrophin expression with vivo-morpholino-mediated multiple exon skipping in dystrophic dogs.
    Yokota T, Nakamura A, Nagata T, Saito T, Kobayashi M, Aoki Y, Echigoya Y, Partridge T, Hoffman EP, Takeda S.
    Nucleic Acid Ther; 2012 Oct 18; 22(5):306-15. PubMed ID: 22888777
    [Abstract] [Full Text] [Related]

  • 40. Creation of DMD Muscle Cell Model Using CRISPR-Cas9 Genome Editing to Test the Efficacy of Antisense-Mediated Exon Skipping.
    Maruyama R, Yokota T.
    Methods Mol Biol; 2018 Oct 18; 1828():165-171. PubMed ID: 30171541
    [Abstract] [Full Text] [Related]

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