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Journal Abstract Search

826 related items for PubMed ID: 31230718

  • 1. 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
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  • 2. Long-Term SMN- and Ncald-ASO Combinatorial Therapy in SMA Mice and NCALD-ASO Treatment in hiPSC-Derived Motor Neurons Show Protective Effects.
    Muiños-Bühl A, Rombo R, Ling KK, Zilio E, Rigo F, Bennett CF, Wirth B.
    Int J Mol Sci; 2023 Feb 20; 24(4):. PubMed ID: 36835624
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  • 7. Evaluation of potential effects of Plastin 3 overexpression and low-dose SMN-antisense oligonucleotides on putative biomarkers in spinal muscular atrophy mice.
    Strathmann EA, Peters M, Hosseinibarkooie S, Rigo FW, Bennett CF, Zaworski PG, Chen KS, Nothnagel M, Wirth B.
    PLoS One; 2018 Feb 20; 13(9):e0203398. PubMed ID: 30188931
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  • 10. The Power of Human Protective Modifiers: PLS3 and CORO1C Unravel Impaired Endocytosis in Spinal Muscular Atrophy and Rescue SMA Phenotype.
    Hosseinibarkooie S, Peters M, Torres-Benito L, Rastetter RH, Hupperich K, Hoffmann A, Mendoza-Ferreira N, Kaczmarek A, Janzen E, Milbradt J, Lamkemeyer T, Rigo F, Bennett CF, Guschlbauer C, Büschges A, Hammerschmidt M, Riessland M, Kye MJ, Clemen CS, Wirth B.
    Am J Hum Genet; 2016 Sep 01; 99(3):647-665. PubMed ID: 27499521
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  • 11. Nusinersen ameliorates motor function and prevents motoneuron Cajal body disassembly and abnormal poly(A) RNA distribution in a SMA mouse model.
    Berciano MT, Puente-Bedia A, Medina-Samamé A, Rodríguez-Rey JC, Calderó J, Lafarga M, Tapia O.
    Sci Rep; 2020 Jul 01; 10(1):10738. PubMed ID: 32612161
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  • 13. 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
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  • 15. 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 Mar 02; 11(12):e0167077. PubMed ID: 27907033
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  • 17. Nusinersen: A Review in 5q Spinal Muscular Atrophy.
    Hoy SM.
    CNS Drugs; 2021 Dec 02; 35(12):1317-1328. PubMed ID: 34850360
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  • 18. Antisense oligonucleotides targeting the SMN2 promoter region enhance SMN2 expression in spinal muscular atrophy cell lines and mouse model.
    Wang J, Bai J, OuYang S, Wang H, Jin Y, Peng X, Ge X, Jiao H, Zou J, He C, Xiao P, Song F, Qu Y.
    Hum Mol Genet; 2022 May 19; 31(10):1635-1650. PubMed ID: 34888619
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  • 20. A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse.
    Porensky PN, Mitrpant C, McGovern VL, Bevan AK, Foust KD, Kaspar BK, Wilton SD, Burghes AH.
    Hum Mol Genet; 2012 Apr 01; 21(7):1625-38. PubMed ID: 22186025
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