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Journal Abstract Search

506 related items for PubMed ID: 31873063

  • 1. Systemic Evaluation of Chimeric LNA/2'-O-Methyl Steric Blockers for Myotonic Dystrophy Type 1 Therapy.
    Christou M, Wengel J, Sokratous K, Kyriacou K, Nikolaou G, Phylactou LA, Mastroyiannopoulos NP.
    Nucleic Acid Ther; 2020 Apr; 30(2):80-93. PubMed ID: 31873063
    [Abstract] [Full Text] [Related]

  • 2. Systemic therapy in an RNA toxicity mouse model with an antisense oligonucleotide therapy targeting a non-CUG sequence within the DMPK 3'UTR RNA.
    Yadava RS, Yu Q, Mandal M, Rigo F, Bennett CF, Mahadevan MS.
    Hum Mol Genet; 2020 Jun 03; 29(9):1440-1453. PubMed ID: 32242217
    [Abstract] [Full Text] [Related]

  • 3. Short antisense-locked nucleic acids (all-LNAs) correct alternative splicing abnormalities in myotonic dystrophy.
    Wojtkowiak-Szlachcic A, Taylor K, Stepniak-Konieczna E, Sznajder LJ, Mykowska A, Sroka J, Thornton CA, Sobczak K.
    Nucleic Acids Res; 2015 Mar 31; 43(6):3318-31. PubMed ID: 25753670
    [Abstract] [Full Text] [Related]

  • 4. Systemic delivery of a Peptide-linked morpholino oligonucleotide neutralizes mutant RNA toxicity in a mouse model of myotonic dystrophy.
    Leger AJ, Mosquea LM, Clayton NP, Wu IH, Weeden T, Nelson CA, Phillips L, Roberts E, Piepenhagen PA, Cheng SH, Wentworth BM.
    Nucleic Acid Ther; 2013 Apr 31; 23(2):109-17. PubMed ID: 23308382
    [Abstract] [Full Text] [Related]

  • 5. Muscleblind-like 1 knockout mice reveal novel splicing defects in the myotonic dystrophy brain.
    Suenaga K, Lee KY, Nakamori M, Tatsumi Y, Takahashi MP, Fujimura H, Jinnai K, Yoshikawa H, Du H, Ares M, Swanson MS, Kimura T.
    PLoS One; 2012 Apr 31; 7(3):e33218. PubMed ID: 22427994
    [Abstract] [Full Text] [Related]

  • 6. Molecular mechanisms responsible for aberrant splicing of SERCA1 in myotonic dystrophy type 1.
    Hino S, Kondo S, Sekiya H, Saito A, Kanemoto S, Murakami T, Chihara K, Aoki Y, Nakamori M, Takahashi MP, Imaizumi K.
    Hum Mol Genet; 2007 Dec 01; 16(23):2834-43. PubMed ID: 17728322
    [Abstract] [Full Text] [Related]

  • 7. Reduced cytoplasmic MBNL1 is an early event in a brain-specific mouse model of myotonic dystrophy.
    Wang PY, Lin YM, Wang LH, Kuo TY, Cheng SJ, Wang GS.
    Hum Mol Genet; 2017 Jun 15; 26(12):2247-2257. PubMed ID: 28369378
    [Abstract] [Full Text] [Related]

  • 8. Fuchs' Endothelial Corneal Dystrophy and RNA Foci in Patients With Myotonic Dystrophy.
    Mootha VV, Hansen B, Rong Z, Mammen PP, Zhou Z, Xing C, Gong X.
    Invest Ophthalmol Vis Sci; 2017 Sep 01; 58(11):4579-4585. PubMed ID: 28886202
    [Abstract] [Full Text] [Related]

  • 9. In silico discovery of substituted pyrido[2,3-d]pyrimidines and pentamidine-like compounds with biological activity in myotonic dystrophy models.
    González ÀL, Konieczny P, Llamusi B, Delgado-Pinar E, Borrell JI, Teixidó J, García-España E, Pérez-Alonso M, Estrada-Tejedor R, Artero R.
    PLoS One; 2017 Sep 01; 12(6):e0178931. PubMed ID: 28582438
    [Abstract] [Full Text] [Related]

  • 10. AON-induced splice-switching and DMPK pre-mRNA degradation as potential therapeutic approaches for Myotonic Dystrophy type 1.
    Stepniak-Konieczna E, Konieczny P, Cywoniuk P, Dluzewska J, Sobczak K.
    Nucleic Acids Res; 2020 Mar 18; 48(5):2531-2543. PubMed ID: 31965181
    [Abstract] [Full Text] [Related]

  • 11. RNA interference targeting CUG repeats in a mouse model of myotonic dystrophy.
    Sobczak K, Wheeler TM, Wang W, Thornton CA.
    Mol Ther; 2013 Feb 18; 21(2):380-7. PubMed ID: 23183533
    [Abstract] [Full Text] [Related]

  • 12. A flow cytometry-based screen identifies MBNL1 modulators that rescue splicing defects in myotonic dystrophy type I.
    Zhang F, Bodycombe NE, Haskell KM, Sun YL, Wang ET, Morris CA, Jones LH, Wood LD, Pletcher MT.
    Hum Mol Genet; 2017 Aug 15; 26(16):3056-3068. PubMed ID: 28535287
    [Abstract] [Full Text] [Related]

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  • 14. (CTG)n repeat-mediated dysregulation of MBNL1 and MBNL2 expression during myogenesis in DM1 occurs already at the myoblast stage.
    André LM, van Cruchten RTP, Willemse M, Wansink DG.
    PLoS One; 2019 Aug 15; 14(5):e0217317. PubMed ID: 31116797
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  • 19. Recent Progress and Challenges in the Development of Antisense Therapies for Myotonic Dystrophy Type 1.
    De Serres-Bérard T, Ait Benichou S, Jauvin D, Boutjdir M, Puymirat J, Chahine M.
    Int J Mol Sci; 2022 Nov 01; 23(21):. PubMed ID: 36362145
    [Abstract] [Full Text] [Related]

  • 20. Expanded CUG repeats in DMPK transcripts adopt diverse hairpin conformations without influencing the structure of the flanking sequences.
    van Cruchten RTP, Wieringa B, Wansink DG.
    RNA; 2019 Apr 01; 25(4):481-495. PubMed ID: 30700578
    [Abstract] [Full Text] [Related]

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