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Journal Abstract Search

296 related items for PubMed ID: 27163330

  • 1. 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; 11(5):e0155032. PubMed ID: 27163330
    [Abstract] [Full Text] [Related]

  • 2. 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]

  • 3. Repeated low doses of morpholino antisense oligomer: an intermediate mouse model of spinal muscular atrophy to explore the window of therapeutic response.
    Zhou H, Meng J, Marrosu E, Janghra N, Morgan J, Muntoni F.
    Hum Mol Genet; 2015 Nov 15; 24(22):6265-77. PubMed ID: 26264577
    [Abstract] [Full Text] [Related]

  • 4. 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 Nov 15; 11(12):e0167077. PubMed ID: 27907033
    [Abstract] [Full Text] [Related]

  • 5. Survival motor neuron protein regulates oxidative stress and inflammatory response in microglia of the spinal cord in spinal muscular atrophy.
    Ando S, Osanai D, Takahashi K, Nakamura S, Shimazawa M, Hara H.
    J Pharmacol Sci; 2020 Dec 15; 144(4):204-211. PubMed ID: 33070839
    [Abstract] [Full Text] [Related]

  • 6. Molecular, genetic and stem cell-mediated therapeutic strategies for spinal muscular atrophy (SMA).
    Zanetta C, Riboldi G, Nizzardo M, Simone C, Faravelli I, Bresolin N, Comi GP, Corti S.
    J Cell Mol Med; 2014 Feb 15; 18(2):187-96. PubMed ID: 24400925
    [Abstract] [Full Text] [Related]

  • 7. Selective Neuromuscular Denervation in Taiwanese Severe SMA Mouse Can Be Reversed by Morpholino Antisense Oligonucleotides.
    Lin TL, Chen TH, Hsu YY, Cheng YH, Juang BT, Jong YJ.
    PLoS One; 2016 Feb 15; 11(4):e0154723. PubMed ID: 27124114
    [Abstract] [Full Text] [Related]

  • 8. 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]

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  • 10. 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]

  • 11. 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]

  • 12. 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]

  • 13. Interaction between alpha-COP and SMN ameliorates disease phenotype in a mouse model of spinal muscular atrophy.
    Custer SK, Astroski JW, Li HX, Androphy EJ.
    Biochem Biophys Res Commun; 2019 Jun 25; 514(2):530-537. PubMed ID: 31060774
    [Abstract] [Full Text] [Related]

  • 14. Muscle-specific SMN reduction reveals motor neuron-independent disease in spinal muscular atrophy models.
    Kim JK, Jha NN, Feng Z, Faleiro MR, Chiriboga CA, Wei-Lapierre L, Dirksen RT, Ko CP, Monani UR.
    J Clin Invest; 2020 Mar 02; 130(3):1271-1287. PubMed ID: 32039917
    [Abstract] [Full Text] [Related]

  • 15. 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]

  • 16. Calpain Inhibition Increases SMN Protein in Spinal Cord Motoneurons and Ameliorates the Spinal Muscular Atrophy Phenotype in Mice.
    de la Fuente S, Sansa A, Periyakaruppiah A, Garcera A, Soler RM.
    Mol Neurobiol; 2019 Jun 01; 56(6):4414-4427. PubMed ID: 30327977
    [Abstract] [Full Text] [Related]

  • 17. Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy.
    Zhou H, Meng J, Malerba A, Catapano F, Sintusek P, Jarmin S, Feng L, Lu-Nguyen N, Sun L, Mariot V, Dumonceaux J, Morgan JE, Gissen P, Dickson G, Muntoni F.
    J Cachexia Sarcopenia Muscle; 2020 Jun 01; 11(3):768-782. PubMed ID: 32031328
    [Abstract] [Full Text] [Related]

  • 18. Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates.
    Meyer K, Ferraiuolo L, Schmelzer L, Braun L, McGovern V, Likhite S, Michels O, Govoni A, Fitzgerald J, Morales P, Foust KD, Mendell JR, Burghes AH, Kaspar BK.
    Mol Ther; 2015 Mar 01; 23(3):477-87. PubMed ID: 25358252
    [Abstract] [Full Text] [Related]

  • 19. SMN deficiency in severe models of spinal muscular atrophy causes widespread intron retention and DNA damage.
    Jangi M, Fleet C, Cullen P, Gupta SV, Mekhoubad S, Chiao E, Allaire N, Bennett CF, Rigo F, Krainer AR, Hurt JA, Carulli JP, Staropoli JF.
    Proc Natl Acad Sci U S A; 2017 Mar 21; 114(12):E2347-E2356. PubMed ID: 28270613
    [Abstract] [Full Text] [Related]

  • 20. 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]

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