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Page 1
Evaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse Models.
Moore LR, Rajpal G, Dillingham IT, Qutob M, Blumenstein KG, Gattis D, Hung G, Kordasiewicz HB, Paulson HL, McLoughlin HS. Moore LR, et al. Among authors: mcloughlin hs. Mol Ther Nucleic Acids. 2017 Jun 16;7:200-210. doi: 10.1016/j.omtn.2017.04.005. Epub 2017 Apr 12. Mol Ther Nucleic Acids. 2017. PMID: 28624196 Free PMC article.
Synthetic high-density lipoprotein nanoparticles for the treatment of Niemann-Pick diseases.
Schultz ML, Fawaz MV, Azaria RD, Hollon TC, Liu EA, Kunkel TJ, Halseth TA, Krus KL, Ming R, Morin EE, McLoughlin HS, Bushart DD, Paulson HL, Shakkottai VG, Orringer DA, Schwendeman AS, Lieberman AP. Schultz ML, et al. Among authors: mcloughlin hs. BMC Med. 2019 Nov 11;17(1):200. doi: 10.1186/s12916-019-1423-5. BMC Med. 2019. PMID: 31711490 Free PMC article.
Toward allele-specific targeting therapy and pharmacodynamic marker for spinocerebellar ataxia type 3.
Prudencio M, Garcia-Moreno H, Jansen-West KR, Al-Shaikh RH, Gendron TF, Heckman MG, Spiegel MR, Carlomagno Y, Daughrity LM, Song Y, Dunmore JA, Byron N, Oskarsson B, Nicholson KA, Staff NP, Gorcenco S, Puschmann A, Lemos J, Januário C, LeDoux MS, Friedman JH, Polke J, Labrum R, Shakkottai V, McLoughlin HS, Paulson HL, Konno T, Onodera O, Ikeuchi T, Tada M, Kakita A, Fryer JD, Karremo C, Gomes I, Caviness JN, Pittelkow MR, Aasly J, Pfeiffer RF, Veerappan V, Eggenberger ER, Freeman WD, Huang JF, Uitti RJ, Wierenga KJ, Marin Collazo IV, Tipton PW, van Gerpen JA, van Blitterswijk M, Bu G, Wszolek ZK, Giunti P, Petrucelli L. Prudencio M, et al. Among authors: mcloughlin hs. Sci Transl Med. 2020 Oct 21;12(566):eabb7086. doi: 10.1126/scitranslmed.abb7086. Sci Transl Med. 2020. PMID: 33087504 Free PMC article.
Impaired Oligodendrocyte Maturation Is an Early Feature in SCA3 Disease Pathogenesis.
Schuster KH, Zalon AJ, Zhang H, DiFranco DM, Stec NR, Haque Z, Blumenstein KG, Pierce AM, Guan Y, Paulson HL, McLoughlin HS. Schuster KH, et al. Among authors: mcloughlin hs. J Neurosci. 2022 Feb 23;42(8):1604-1617. doi: 10.1523/JNEUROSCI.1954-20.2021. Epub 2022 Jan 18. J Neurosci. 2022. PMID: 35042771 Free PMC article.
27 results