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433 related items for PubMed ID: 31363739

  • 1. AAV9-Stathmin1 gene delivery improves disease phenotype in an intermediate mouse model of spinal muscular atrophy.
    Villalón E, Kline RA, Smith CE, Lorson ZC, Osman EY, O'Day S, Murray LM, Lorson CL.
    Hum Mol Genet; 2019 Nov 15; 28(22):3742-3754. PubMed ID: 31363739
    [Abstract] [Full Text] [Related]

  • 2. Decreased stathmin expression ameliorates neuromuscular defects but fails to prolong survival in a mouse model of spinal muscular atrophy.
    Wen HL, Ting CH, Liu HC, Li H, Lin-Chao S.
    Neurobiol Dis; 2013 Apr 15; 52():94-103. PubMed ID: 23268200
    [Abstract] [Full Text] [Related]

  • 3. AAV9-Mediated Expression of SMN Restricted to Neurons Does Not Rescue the Spinal Muscular Atrophy Phenotype in Mice.
    Besse A, Astord S, Marais T, Roda M, Giroux B, Lejeune FX, Relaix F, Smeriglio P, Barkats M, Biferi MG.
    Mol Ther; 2020 Aug 05; 28(8):1887-1901. PubMed ID: 32470325
    [Abstract] [Full Text] [Related]

  • 4. Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice.
    Dominguez E, Marais T, Chatauret N, Benkhelifa-Ziyyat S, Duque S, Ravassard P, Carcenac R, Astord S, Pereira de Moura A, Voit T, Barkats M.
    Hum Mol Genet; 2011 Feb 15; 20(4):681-93. PubMed ID: 21118896
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  • 5. A large animal model of spinal muscular atrophy and correction of phenotype.
    Duque SI, Arnold WD, Odermatt P, Li X, Porensky PN, Schmelzer L, Meyer K, Kolb SJ, Schümperli D, Kaspar BK, Burghes AH.
    Ann Neurol; 2015 Mar 15; 77(3):399-414. PubMed ID: 25516063
    [Abstract] [Full Text] [Related]

  • 6. Central and peripheral delivered AAV9-SMN are both efficient but target different pathomechanisms in a mouse model of spinal muscular atrophy.
    Reilly A, Deguise MO, Beauvais A, Yaworski R, Thebault S, Tessier DR, Tabard-Cossa V, Hensel N, Schneider BL, Kothary R.
    Gene Ther; 2022 Sep 15; 29(9):544-554. PubMed ID: 35462564
    [Abstract] [Full Text] [Related]

  • 7. Improved therapeutic approach for spinal muscular atrophy via ubiquitination-resistant survival motor neuron variant.
    Rhee J, Kang JS, Jo YW, Yoo K, Kim YL, Hann SH, Kim YE, Kim H, Kim JH, Kong YY.
    J Cachexia Sarcopenia Muscle; 2024 Aug 15; 15(4):1404-1417. PubMed ID: 38650097
    [Abstract] [Full Text] [Related]

  • 8. 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 15; 23(3):477-87. PubMed ID: 25358252
    [Abstract] [Full Text] [Related]

  • 9. Stathmin, a microtubule-destabilizing protein, is dysregulated in spinal muscular atrophy.
    Wen HL, Lin YT, Ting CH, Lin-Chao S, Li H, Hsieh-Li HM.
    Hum Mol Genet; 2010 May 01; 19(9):1766-78. PubMed ID: 20176735
    [Abstract] [Full Text] [Related]

  • 10. Astrocytes influence the severity of spinal muscular atrophy.
    Rindt H, Feng Z, Mazzasette C, Glascock JJ, Valdivia D, Pyles N, Crawford TO, Swoboda KJ, Patitucci TN, Ebert AD, Sumner CJ, Ko CP, Lorson CL.
    Hum Mol Genet; 2015 Jul 15; 24(14):4094-102. PubMed ID: 25911676
    [Abstract] [Full Text] [Related]

  • 11. Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN.
    Foust KD, Wang X, McGovern VL, Braun L, Bevan AK, Haidet AM, Le TT, Morales PR, Rich MM, Burghes AH, Kaspar BK.
    Nat Biotechnol; 2010 Mar 15; 28(3):271-4. PubMed ID: 20190738
    [Abstract] [Full Text] [Related]

  • 12. Motor transmission defects with sex differences in a new mouse model of mild spinal muscular atrophy.
    Deguise MO, De Repentigny Y, Tierney A, Beauvais A, Michaud J, Chehade L, Thabet M, Paul B, Reilly A, Gagnon S, Renaud JM, Kothary R.
    EBioMedicine; 2020 May 15; 55():102750. PubMed ID: 32339936
    [Abstract] [Full Text] [Related]

  • 13. Translational fidelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy.
    Passini MA, Bu J, Richards AM, Treleaven CM, Sullivan JA, O'Riordan CR, Scaria A, Kells AP, Samaranch L, San Sebastian W, Federici T, Fiandaca MS, Boulis NM, Bankiewicz KS, Shihabuddin LS, Cheng SH.
    Hum Gene Ther; 2014 Jul 15; 25(7):619-30. PubMed ID: 24617515
    [Abstract] [Full Text] [Related]

  • 14. Pharmacologically induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset.
    Feng Z, Ling KK, Zhao X, Zhou C, Karp G, Welch EM, Naryshkin N, Ratni H, Chen KS, Metzger F, Paushkin S, Weetall M, Ko CP.
    Hum Mol Genet; 2016 Mar 01; 25(5):964-75. PubMed ID: 26758873
    [Abstract] [Full Text] [Related]

  • 15. Defining the therapeutic window in a severe animal model of spinal muscular atrophy.
    Robbins KL, Glascock JJ, Osman EY, Miller MR, Lorson CL.
    Hum Mol Genet; 2014 Sep 01; 23(17):4559-68. PubMed ID: 24722206
    [Abstract] [Full Text] [Related]

  • 16. CHP1 reduction ameliorates spinal muscular atrophy pathology by restoring calcineurin activity and endocytosis.
    Janzen E, Mendoza-Ferreira N, Hosseinibarkooie S, Schneider S, Hupperich K, Tschanz T, Grysko V, Riessland M, Hammerschmidt M, Rigo F, Bennett CF, Kye MJ, Torres-Benito L, Wirth B.
    Brain; 2018 Aug 01; 141(8):2343-2361. PubMed ID: 29961886
    [Abstract] [Full Text] [Related]

  • 17. Early heart failure in the SMNDelta7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery.
    Bevan AK, Hutchinson KR, Foust KD, Braun L, McGovern VL, Schmelzer L, Ward JG, Petruska JC, Lucchesi PA, Burghes AH, Kaspar BK.
    Hum Mol Genet; 2010 Oct 15; 19(20):3895-905. PubMed ID: 20639395
    [Abstract] [Full Text] [Related]

  • 18. PTEN depletion decreases disease severity and modestly prolongs survival in a mouse model of spinal muscular atrophy.
    Little D, Valori CF, Mutsaers CA, Bennett EJ, Wyles M, Sharrack B, Shaw PJ, Gillingwater TH, Azzouz M, Ning K.
    Mol Ther; 2015 Feb 15; 23(2):270-7. PubMed ID: 25369768
    [Abstract] [Full Text] [Related]

  • 19. A critical smn threshold in mice dictates onset of an intermediate spinal muscular atrophy phenotype associated with a distinct neuromuscular junction pathology.
    Bowerman M, Murray LM, Beauvais A, Pinheiro B, Kothary R.
    Neuromuscul Disord; 2012 Mar 15; 22(3):263-76. PubMed ID: 22071333
    [Abstract] [Full Text] [Related]

  • 20. Limited phenotypic effects of selectively augmenting the SMN protein in the neurons of a mouse model of severe spinal muscular atrophy.
    Lee AJ, Awano T, Park GH, Monani UR.
    PLoS One; 2012 Mar 15; 7(9):e46353. PubMed ID: 23029491
    [Abstract] [Full Text] [Related]

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