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Journal Abstract Search

209 related items for PubMed ID: 36835624

  • 1. 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
    [Abstract] [Full Text] [Related]

  • 2. 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]

  • 3. Combinatorial ASO-mediated therapy with low dose SMN and the protective modifier Chp1 is not sufficient to ameliorate SMA pathology hallmarks.
    Muinos-Bühl A, Rombo R, Janzen E, Ling KK, Hupperich K, Rigo F, Bennett CF, Wirth B.
    Neurobiol Dis; 2022 Sep 03; 171():105795. PubMed ID: 35724821
    [Abstract] [Full Text] [Related]

  • 4. Neurocalcin Delta Suppression Protects against Spinal Muscular Atrophy in Humans and across Species by Restoring Impaired Endocytosis.
    Riessland M, Kaczmarek A, Schneider S, Swoboda KJ, Löhr H, Bradler C, Grysko V, Dimitriadi M, Hosseinibarkooie S, Torres-Benito L, Peters M, Upadhyay A, Biglari N, Kröber S, Hölker I, Garbes L, Gilissen C, Hoischen A, Nürnberg G, Nürnberg P, Walter M, Rigo F, Bennett CF, Kye MJ, Hart AC, Hammerschmidt M, Kloppenburg P, Wirth B.
    Am J Hum Genet; 2017 Feb 02; 100(2):297-315. PubMed ID: 28132687
    [Abstract] [Full Text] [Related]

  • 5. 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]

  • 6. 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
    [Abstract] [Full Text] [Related]

  • 7. 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]

  • 8. 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 Oct 05; 13(9):e0203398. PubMed ID: 30188931
    [Abstract] [Full Text] [Related]

  • 9. 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 Oct 05; 11(12):e0167077. PubMed ID: 27907033
    [Abstract] [Full Text] [Related]

  • 10. The Antisense Transcript SMN-AS1 Regulates SMN Expression and Is a Novel Therapeutic Target for Spinal Muscular Atrophy.
    d'Ydewalle C, Ramos DM, Pyles NJ, Ng SY, Gorz M, Pilato CM, Ling K, Kong L, Ward AJ, Rubin LL, Rigo F, Bennett CF, Sumner CJ.
    Neuron; 2017 Jan 04; 93(1):66-79. PubMed ID: 28017471
    [Abstract] [Full Text] [Related]

  • 11. Combined treatment with the histone deacetylase inhibitor LBH589 and a splice-switch antisense oligonucleotide enhances SMN2 splicing and SMN expression in Spinal Muscular Atrophy cells.
    Pagliarini V, Guerra M, Di Rosa V, Compagnucci C, Sette C.
    J Neurochem; 2020 Apr 04; 153(2):264-275. PubMed ID: 31811660
    [Abstract] [Full Text] [Related]

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  • 15. A combinatorial approach increases SMN level in SMA model mice.
    Dumas SA, Villalón E, Bergman EM, Wilson KJ, Marugan JJ, Lorson CL, Burnett BG.
    Hum Mol Genet; 2022 Aug 25; 31(17):2989-3000. PubMed ID: 35419606
    [Abstract] [Full Text] [Related]

  • 16. The human centromeric survival motor neuron gene (SMN2) rescues embryonic lethality in Smn(-/-) mice and results in a mouse with spinal muscular atrophy.
    Monani UR, Sendtner M, Coovert DD, Parsons DW, Andreassi C, Le TT, Jablonka S, Schrank B, Rossoll W, Prior TW, Morris GE, Burghes AH.
    Hum Mol Genet; 2000 Feb 12; 9(3):333-9. PubMed ID: 10655541
    [Abstract] [Full Text] [Related]

  • 17. Motor unit recovery following Smn restoration in mouse models of spinal muscular atrophy.
    Comley LH, Kline RA, Thomson AK, Woschitz V, Landeros EV, Osman EY, Lorson CL, Murray LM.
    Hum Mol Genet; 2022 Sep 10; 31(18):3107-3119. PubMed ID: 35551393
    [Abstract] [Full Text] [Related]

  • 18. 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]

  • 19. 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]

  • 20. Spinal muscular atrophy phenotype is ameliorated in human motor neurons by SMN increase via different novel RNA therapeutic approaches.
    Nizzardo M, Simone C, Dametti S, Salani S, Ulzi G, Pagliarani S, Rizzo F, Frattini E, Pagani F, Bresolin N, Comi G, Corti S.
    Sci Rep; 2015 Jun 30; 5():11746. PubMed ID: 26123042
    [Abstract] [Full Text] [Related]

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