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Page 1
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: daldin m. 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
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. Sci Rep. 2017 Jul 11;7(1):5070. doi: 10.1038/s41598-017-05336-7. Sci Rep. 2017. PMID: 28698602 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: daldin m. PLoS One. 2014 Dec 2;9(12):e112262. doi: 10.1371/journal.pone.0112262. eCollection 2014. PLoS One. 2014. PMID: 25464275 Free PMC article.