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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: heikkinen tt. 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
The PDE1/5 Inhibitor SCH-51866 Does Not Modify Disease Progression in the R6/2 Mouse Model of Huntington's Disease.
Beaumont V, Park L, Rassoulpour A, Dijkman U, Heikkinen T, Lehtimaki K, Kontkanen O, Al Nackkash R, Bates GP, Gleyzes M, Steidl E, Ramboz S, Murphy C, Beconi MG, Dominguez C, Munoz-Sanjuan I. Beaumont V, et al. PLoS Curr. 2014 Feb 13;6:ecurrents.hd.3304e87e460b4bb0dc519a29f4deccca. doi: 10.1371/currents.hd.3304e87e460b4bb0dc519a29f4deccca. PLoS Curr. 2014. PMID: 24558637 Free PMC article.
The novel KMO inhibitor CHDI-340246 leads to a restoration of electrophysiological alterations in mouse models of Huntington's disease.
Beaumont V, Mrzljak L, Dijkman U, Freije R, Heins M, Rassoulpour A, Tombaugh G, Gelman S, Bradaia A, Steidl E, Gleyzes M, Heikkinen T, Lehtimäki K, Puoliväli J, Kontkanen O, Javier RM, Neagoe I, Deisemann H, Winkler D, Ebneth A, Khetarpal V, Toledo-Sherman L, Dominguez C, Park LC, Munoz-Sanjuan I. Beaumont V, et al. Exp Neurol. 2016 Aug;282:99-118. doi: 10.1016/j.expneurol.2016.05.005. Epub 2016 May 6. Exp Neurol. 2016. PMID: 27163548
Global Rhes knockout in the Q175 Huntington's disease mouse model.
Heikkinen T, Bragge T, Kuosmanen J, Parkkari T, Gustafsson S, Kwan M, Beltran J, Ghavami A, Subramaniam S, Shahani N, Ramírez-Jarquín UN, Park L, Muñoz-Sanjuán I, Marchionini DM. Heikkinen T, et al. PLoS One. 2021 Oct 14;16(10):e0258486. doi: 10.1371/journal.pone.0258486. eCollection 2021. PLoS One. 2021. PMID: 34648564 Free PMC article.
Characterization of neurophysiological and behavioral changes, MRI brain volumetry and 1H MRS in zQ175 knock-in mouse model of Huntington's disease.
Heikkinen T, Lehtimäki K, Vartiainen N, Puoliväli J, Hendricks SJ, Glaser JR, Bradaia A, Wadel K, Touller C, Kontkanen O, Yrjänheikki JM, Buisson B, Howland D, Beaumont V, Munoz-Sanjuan I, Park LC. Heikkinen T, et al. PLoS One. 2012;7(12):e50717. doi: 10.1371/journal.pone.0050717. Epub 2012 Dec 20. PLoS One. 2012. PMID: 23284644 Free PMC article.
Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington's disease.
Zeitler B, Froelich S, Marlen K, Shivak DA, Yu Q, Li D, Pearl JR, Miller JC, Zhang L, Paschon DE, Hinkley SJ, Ankoudinova I, Lam S, Guschin D, Kopan L, Cherone JM, Nguyen HB, Qiao G, Ataei Y, Mendel MC, Amora R, Surosky R, Laganiere J, Vu BJ, Narayanan A, Sedaghat Y, Tillack K, Thiede C, Gärtner A, Kwak S, Bard J, Mrzljak L, Park L, Heikkinen T, Lehtimäki KK, Svedberg MM, Häggkvist J, Tari L, Tóth M, Varrone A, Halldin C, Kudwa AE, Ramboz S, Day M, Kondapalli J, Surmeier DJ, Urnov FD, Gregory PD, Rebar EJ, Muñoz-Sanjuán I, Zhang HS. Zeitler B, et al. Nat Med. 2019 Jul;25(7):1131-1142. doi: 10.1038/s41591-019-0478-3. Epub 2019 Jul 1. Nat Med. 2019. PMID: 31263285
Phosphodiesterase 10A Inhibition Improves Cortico-Basal Ganglia Function in Huntington's Disease Models.
Beaumont V, Zhong S, Lin H, Xu W, Bradaia A, Steidl E, Gleyzes M, Wadel K, Buisson B, Padovan-Neto FE, Chakroborty S, Ward KM, Harms JF, Beltran J, Kwan M, Ghavami A, Häggkvist J, Tóth M, Halldin C, Varrone A, Schaab C, Dybowski JN, Elschenbroich S, Lehtimäki K, Heikkinen T, Park L, Rosinski J, Mrzljak L, Lavery D, West AR, Schmidt CJ, Zaleska MM, Munoz-Sanjuan I. Beaumont V, et al. Neuron. 2016 Dec 21;92(6):1220-1237. doi: 10.1016/j.neuron.2016.10.064. Epub 2016 Dec 1. Neuron. 2016. PMID: 27916455 Free article.
15 results