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Identification and Optimization of RNA-Splicing Modulators as Huntingtin Protein-Lowering Agents for the Treatment of Huntington's Disease.
Liu L, Malagu K, Haughan AF, Khetarpal V, Stott AJ, Esmieu W, Vater HD, Webster SJ, Van de Poël AJ, Clissold C, Cosgrove B, Sutton B, Spencer JA, Breccia P, Gancia E, Bonomo S, Ladduwahetty T, Lazari O, Patel H, Atton HC, Clifton S, Mota DM, Magnani D, O'Neill A, Stebbeds M, Macabuag N, Todd D, Herva ME, Mitchell P, Visser M, Compte Sancerni S, Grand Moursel L, da Silva M, Kritikou E, Heikkinen TT, Bolkvadze T, Fodale V, Spadafora D, Daldin M, Bresciani A, Mangette JE, Doherty EM, Lee MR, Herbst T, Monteagudo E, Macdonald D, Plotnikov NV, Chambers M, McAllister G, Muňoz-Sanjuan I, Dominguez C. Liu L, et al. Among authors: macdonald d. J Med Chem. 2023 Sep 28;66(18):13205-13246. doi: 10.1021/acs.jmedchem.3c01173. Epub 2023 Sep 15. J Med Chem. 2023. PMID: 37712656
Discovery and structure-activity relationship of potent and selective covalent inhibitors of transglutaminase 2 for Huntington's disease.
Prime ME, Andersen OA, Barker JJ, Brooks MA, Cheng RK, Toogood-Johnson I, Courtney SM, Brookfield FA, Yarnold CJ, Marston RW, Johnson PD, Johnsen SF, Palfrey JJ, Vaidya D, Erfan S, Ichihara O, Felicetti B, Palan S, Pedret-Dunn A, Schaertl S, Sternberger I, Ebneth A, Scheel A, Winkler D, Toledo-Sherman L, Beconi M, Macdonald D, Muñoz-Sanjuan I, Dominguez C, Wityak J. Prime ME, et al. Among authors: macdonald d. J Med Chem. 2012 Feb 9;55(3):1021-46. doi: 10.1021/jm201310y. Epub 2012 Jan 27. J Med Chem. 2012. PMID: 22224594
Quantification assays for total and polyglutamine-expanded huntingtin proteins.
Macdonald D, Tessari MA, Boogaard I, Smith M, Pulli K, Szynol A, Albertus F, Lamers MB, Dijkstra S, Kordt D, Reindl W, Herrmann F, McAllister G, Fischer DF, Munoz-Sanjuan I. Macdonald D, et al. PLoS One. 2014 May 9;9(5):e96854. doi: 10.1371/journal.pone.0096854. eCollection 2014. PLoS One. 2014. PMID: 24816435 Free PMC article.
SAR Development of Lysine-Based Irreversible Inhibitors of Transglutaminase 2 for Huntington's Disease.
Wityak J, Prime ME, Brookfield FA, Courtney SM, Erfan S, Johnsen S, Johnson PD, Li M, Marston RW, Reed L, Vaidya D, Schaertl S, Pedret-Dunn A, Beconi M, Macdonald D, Muñoz-Sanjuan I, Dominguez C. Wityak J, et al. Among authors: macdonald d. ACS Med Chem Lett. 2012 Oct 4;3(12):1024-8. doi: 10.1021/ml300241m. eCollection 2012 Dec 13. ACS Med Chem Lett. 2012. PMID: 24900424 Free PMC article.
Irreversible 4-Aminopiperidine Transglutaminase 2 Inhibitors for Huntington's Disease.
Prime ME, Brookfield FA, Courtney SM, Gaines S, Marston RW, Ichihara O, Li M, Vaidya D, Williams H, Pedret-Dunn A, Reed L, Schaertl S, Toledo-Sherman L, Beconi M, Macdonald D, Muñoz-Sanjuan I, Dominguez C, Wityak J. Prime ME, et al. Among authors: macdonald d. ACS Med Chem Lett. 2012 Aug 9;3(9):731-5. doi: 10.1021/ml3001352. eCollection 2012 Sep 13. ACS Med Chem Lett. 2012. PMID: 24900540 Free PMC article.
Genetic deletion of transglutaminase 2 does not rescue the phenotypic deficits observed in R6/2 and zQ175 mouse models of Huntington's disease.
Menalled LB, Kudwa AE, Oakeshott S, Farrar A, Paterson N, Filippov I, Miller S, Kwan M, Olsen M, Beltran J, Torello J, Fitzpatrick J, Mushlin R, Cox K, McConnell K, Mazzella M, He D, Osborne GF, Al-Nackkash R, Bates GP, Tuunanen P, Lehtimaki K, Brunner D, Ghavami A, Ramboz S, Park L, Macdonald D, Munoz-Sanjuan I, Howland D. Menalled LB, et al. Among authors: macdonald d. PLoS One. 2014 Jun 23;9(6):e99520. doi: 10.1371/journal.pone.0099520. eCollection 2014. PLoS One. 2014. PMID: 24955833 Free PMC article.
Polyglutamine- and temperature-dependent conformational rigidity in mutant huntingtin revealed by immunoassays and circular dichroism spectroscopy.
Fodale V, Kegulian NC, Verani M, Cariulo C, Azzollini L, Petricca L, Daldin M, Boggio R, Padova A, Kuhn R, Pacifici R, Macdonald D, Schoenfeld RC, Park H, Isas JM, Langen R, Weiss A, Caricasole A. Fodale V, et al. Among authors: macdonald d. PLoS One. 2014 Dec 2;9(12):e112262. doi: 10.1371/journal.pone.0112262. eCollection 2014. PLoS One. 2014. PMID: 25464275 Free PMC article.
Polyglutamine expansion affects huntingtin conformation in multiple Huntington's disease models.
Daldin M, Fodale V, Cariulo C, Azzollini L, Verani M, Martufi P, Spiezia MC, Deguire SM, Cherubini M, Macdonald D, Weiss A, Bresciani A, Vonsattel JG, Petricca L, Marsh JL, Gines S, Santimone I, Marano M, Lashuel HA, Squitieri F, Caricasole A. Daldin M, et al. Among authors: macdonald d. Sci Rep. 2017 Jul 11;7(1):5070. doi: 10.1038/s41598-017-05336-7. Sci Rep. 2017. PMID: 28698602 Free PMC article.
2,404 results