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Journal Abstract Search

472 related items for PubMed ID: 24617515

  • 1. 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; 25(7):619-30. PubMed ID: 24617515
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  • 2. 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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  • 3. 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
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  • 4. Intramuscular scAAV9-SMN injection mediates widespread gene delivery to the spinal cord and decreases disease severity in SMA mice.
    Benkhelifa-Ziyyat S, Besse A, Roda M, Duque S, Astord S, Carcenac R, Marais T, Barkats M.
    Mol Ther; 2013 Feb 15; 21(2):282-90. PubMed ID: 23295949
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  • 6. 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
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  • 7. CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy.
    Passini MA, Bu J, Roskelley EM, Richards AM, Sardi SP, O'Riordan CR, Klinger KW, Shihabuddin LS, Cheng SH.
    J Clin Invest; 2010 Apr 15; 120(4):1253-64. PubMed ID: 20234094
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  • 10. 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
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  • 14. 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 Sep 15; 11(12):e0167077. PubMed ID: 27907033
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  • 15. Direct central nervous system delivery provides enhanced protection following vector mediated gene replacement in a severe model of spinal muscular atrophy.
    Glascock JJ, Shababi M, Wetz MJ, Krogman MM, Lorson CL.
    Biochem Biophys Res Commun; 2012 Jan 06; 417(1):376-81. PubMed ID: 22172949
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  • 19. Restoring Bcl-x(L) levels benefits a mouse model of spinal muscular atrophy.
    Tsai LK, Tsai MS, Ting CH, Wang SH, Li H.
    Neurobiol Dis; 2008 Sep 06; 31(3):361-7. PubMed ID: 18590823
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  • 20. SMN deficiency alters Nrxn2 expression and splicing in zebrafish and mouse models of spinal muscular atrophy.
    See K, Yadav P, Giegerich M, Cheong PS, Graf M, Vyas H, Lee SG, Mathavan S, Fischer U, Sendtner M, Winkler C.
    Hum Mol Genet; 2014 Apr 01; 23(7):1754-70. PubMed ID: 24218366
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