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Ataxin-3 protein and RNA toxicity in spinocerebellar ataxia type 3: current insights and emerging therapeutic strategies.
Evers MM, Toonen LJ, van Roon-Mom WM. Evers MM, et al. Mol Neurobiol. 2014 Jun;49(3):1513-31. doi: 10.1007/s12035-013-8596-2. Epub 2013 Nov 29. Mol Neurobiol. 2014. PMID: 24293103 Free PMC article. Review.
Antisense-mediated RNA targeting: versatile and expedient genetic manipulation in the brain.
Zalachoras I, Evers MM, van Roon-Mom WM, Aartsma-Rus AM, Meijer OC. Zalachoras I, et al. Front Mol Neurosci. 2011 Jul 19;4:10. doi: 10.3389/fnmol.2011.00010. eCollection 2011. Front Mol Neurosci. 2011. PMID: 21811437 Free PMC article.
Targeting several CAG expansion diseases by a single antisense oligonucleotide.
Evers MM, Pepers BA, van Deutekom JC, Mulders SA, den Dunnen JT, Aartsma-Rus A, van Ommen GJ, van Roon-Mom WM. Evers MM, et al. PLoS One. 2011;6(9):e24308. doi: 10.1371/journal.pone.0024308. Epub 2011 Sep 1. PLoS One. 2011. PMID: 21909428 Free PMC article.
Ataxin-3 protein modification as a treatment strategy for spinocerebellar ataxia type 3: removal of the CAG containing exon.
Evers MM, Tran HD, Zalachoras I, Pepers BA, Meijer OC, den Dunnen JT, van Ommen GJ, Aartsma-Rus A, van Roon-Mom WM. Evers MM, et al. Neurobiol Dis. 2013 Oct;58:49-56. doi: 10.1016/j.nbd.2013.04.019. Epub 2013 May 6. Neurobiol Dis. 2013. PMID: 23659897
Preventing formation of toxic N-terminal huntingtin fragments through antisense oligonucleotide-mediated protein modification.
Evers MM, Tran HD, Zalachoras I, Meijer OC, den Dunnen JT, van Ommen GJ, Aartsma-Rus A, van Roon-Mom WM. Evers MM, et al. Nucleic Acid Ther. 2014 Feb;24(1):4-12. doi: 10.1089/nat.2013.0452. Epub 2013 Dec 31. Nucleic Acid Ther. 2014. PMID: 24380395
Antisense oligonucleotides in therapy for neurodegenerative disorders.
Evers MM, Toonen LJ, van Roon-Mom WM. Evers MM, et al. Adv Drug Deliv Rev. 2015 Jun 29;87:90-103. doi: 10.1016/j.addr.2015.03.008. Epub 2015 Mar 20. Adv Drug Deliv Rev. 2015. PMID: 25797014 Review.
Making (anti-) sense out of huntingtin levels in Huntington disease.
Evers MM, Schut MH, Pepers BA, Atalar M, van Belzen MJ, Faull RL, Roos RA, van Roon-Mom WM. Evers MM, et al. Mol Neurodegener. 2015 Apr 28;10:21. doi: 10.1186/s13024-015-0018-7. Mol Neurodegener. 2015. PMID: 25928884 Free PMC article.
In vivo proof-of-concept of removal of the huntingtin caspase cleavage motif-encoding exon 12 approach in the YAC128 mouse model of Huntington's disease.
Casaca-Carreira J, Toonen LJA, Evers MM, Jahanshahi A, van-Roon-Mom WMC, Temel Y. Casaca-Carreira J, et al. Biomed Pharmacother. 2016 Dec;84:93-96. doi: 10.1016/j.biopha.2016.09.007. Epub 2016 Sep 16. Biomed Pharmacother. 2016. PMID: 27639545
Transcriptional profiling and biomarker identification reveal tissue specific effects of expanded ataxin-3 in a spinocerebellar ataxia type 3 mouse model.
Toonen LJA, Overzier M, Evers MM, Leon LG, van der Zeeuw SAJ, Mei H, Kielbasa SM, Goeman JJ, Hettne KM, Magnusson OT, Poirel M, Seyer A, 't Hoen PAC, van Roon-Mom WMC. Toonen LJA, et al. Among authors: evers mm. Mol Neurodegener. 2018 Jun 22;13(1):31. doi: 10.1186/s13024-018-0261-9. Mol Neurodegener. 2018. PMID: 29929540 Free PMC article.
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