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Journal Abstract Search

402 related items for PubMed ID: 35724821

  • 1. 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; 171():105795. PubMed ID: 35724821
    [Abstract] [Full Text] [Related]

  • 2. 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]

  • 3. 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]

  • 4. 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]

  • 5. 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
    [Abstract] [Full Text] [Related]

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

  • 7. 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]

  • 8. 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 02; 153(2):264-275. PubMed ID: 31811660
    [Abstract] [Full Text] [Related]

  • 9. Restoring SMN Expression: An Overview of the Therapeutic Developments for the Treatment of Spinal Muscular Atrophy.
    Aslesh T, Yokota T.
    Cells; 2022 Jan 26; 11(3):. PubMed ID: 35159227
    [Abstract] [Full Text] [Related]

  • 10. Recent Progress in Gene-Targeting Therapies for Spinal Muscular Atrophy: Promises and Challenges.
    Haque US, Yokota T.
    Genes (Basel); 2024 Jul 30; 15(8):. PubMed ID: 39202360
    [Abstract] [Full Text] [Related]

  • 11. 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]

  • 12. 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]

  • 13. New and Developing Therapies in Spinal Muscular Atrophy: From Genotype to Phenotype to Treatment and Where Do We Stand?
    Chen TH.
    Int J Mol Sci; 2020 May 07; 21(9):. PubMed ID: 32392694
    [Abstract] [Full Text] [Related]

  • 14. 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]

  • 15. 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]

  • 16. Plastin 3 Expression Does Not Modify Spinal Muscular Atrophy Severity in the ∆7 SMA Mouse.
    McGovern VL, Massoni-Laporte A, Wang X, Le TT, Le HT, Beattie CE, Rich MM, Burghes AH.
    PLoS One; 2015 Oct 05; 10(7):e0132364. PubMed ID: 26134627
    [Abstract] [Full Text] [Related]

  • 17. 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]

  • 18. Dual SMN inducing therapies can rescue survival and motor unit function in symptomatic ∆7SMA mice.
    Kray KM, McGovern VL, Chugh D, Arnold WD, Burghes AHM.
    Neurobiol Dis; 2021 Nov 05; 159():105488. PubMed ID: 34425216
    [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. 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
    [Abstract] [Full Text] [Related]

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