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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.
Mutant huntingtin activates Nrf2-responsive genes and impairs dopamine synthesis in a PC12 model of Huntington's disease.
van Roon-Mom WM, Pepers BA, 't Hoen PA, Verwijmeren CA, den Dunnen JT, Dorsman JC, van Ommen GB. van Roon-Mom WM, et al. BMC Mol Biol. 2008 Oct 9;9:84. doi: 10.1186/1471-2199-9-84. BMC Mol Biol. 2008. PMID: 18844975 Free PMC article.
In silico discovery and experimental validation of new protein-protein interactions.
van Haagen HH, 't Hoen PA, de Morrée A, van Roon-Mom WM, Peters DJ, Roos M, Mons B, van Ommen GJ, Schuemie MJ. van Haagen HH, et al. Proteomics. 2011 Mar;11(5):843-53. doi: 10.1002/pmic.201000398. Epub 2011 Jan 31. Proteomics. 2011. PMID: 21280221
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.
Overview on applications of antisense-mediated exon skipping.
van Roon-Mom WM, Aartsma-Rus A. van Roon-Mom WM, et al. Methods Mol Biol. 2012;867:79-96. doi: 10.1007/978-1-61779-767-5_6. Methods Mol Biol. 2012. PMID: 22454056 Review.
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
Selection and characterization of llama single domain antibodies against N-terminal huntingtin.
Schut MH, Pepers BA, Klooster R, van der Maarel SM, El Khatabi M, Verrips T, den Dunnen JT, van Ommen GJ, van Roon-Mom WM. Schut MH, et al. Neurol Sci. 2015 Mar;36(3):429-34. doi: 10.1007/s10072-014-1971-6. Epub 2014 Oct 8. Neurol Sci. 2015. PMID: 25294428 Free PMC article.
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.
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