Publications

Highlighted Publications

Moore LR, Keller L, Bushart DD, Delatorre RG, Li D, McLoughlin HS, do CarmoCosta M, Shakkottai VG, Smith GD, Paulson HL. Antisense oligonucleotide therapyrescues aggresome formation in a novel spinocerebellar ataxia type 3 humanembryonic stem cell line. Stem Cell Res. 2019 Jul 16;39:101504. doi:10.1016/j.scr.2019.101504. [Epub ahead of print] PubMed PMID: 31374463. Johnson SL, Blount JR, Libohova K, Ranxhi B, Paulson HL, Tsou WL, Todi SV.Differential toxicity of ataxin-3 isoforms in Drosophila models ofSpinocerebellar Ataxia Type 3. Neurobiol Dis. 2019 Jul 13:104535. doi:10.1016/j.nbd.2019.104535. [Epub ahead of print] PubMed PMID: 31310802.Paulson HL, Shakkottai VG, Clark HB, Orr HT. Polyglutamine spinocerebellarataxias - from genes to potential treatments. Nat Rev Neurosci. 2017Oct;18(10):613-626. doi: 10.1038/nrn.2017.92. Epub 2017 Aug 17. Review. PubMedPMID: 28855740; PubMed Central PMCID: PMC6420820. McLoughlin HS, Moore LR, Chopra R, Komlo R, McKenzie M, Blumenstein KG, ZhaoH, Kordasiewicz HB, Shakkottai VG, Paulson HL. Oligonucleotide therapy mitigates disease in spinocerebellar ataxia type 3 mice. Ann Neurol. 2018 Jul;84(1):64-77. doi: 10.1002/ana.25264. Epub 2018 Aug 6. PubMed PMID: 29908063; PubMed CentralPMCID: PMC6119475.
Sharkey LM, Safren N, Pithadia AS, Gerson JE, Dulchavsky M, Fischer S, PatelR, Lantis G, Ashraf N, Kim JH, Meliki A, Minakawa EN, Barmada SJ, Ivanova MI,Paulson HL. Mutant UBQLN2 promotes toxicity by modulating intrinsicself-assembly. Proc Natl Acad Sci U S A. 2018 Oct 30;115(44):E10495-E10504. doi: 10.1073/pnas.1810522115. Epub 2018 Oct 17. PubMed PMID: 30333186; PubMed Central PMCID: PMC6217421.
Ashizawa T, Öz G, Paulson HL. Spinocerebellar ataxias: prospects andchallenges for therapy development. Nat Rev Neurol. 2018 Oct;14(10):590-605. doi:10.1038/s41582-018-0051-6. Review. Erratum in: Nat Rev Neurol. 2018Dec;14(12):749. PubMed PMID: 30131520; PubMed Central PMCID: PMC6469934.

Follow us on social media!