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2015 1
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Decreasing the intrinsically disordered protein α-synuclein levels by targeting its structured mRNA with a ribonuclease-targeting chimera.
Tong Y, Zhang P, Yang X, Liu X, Zhang J, Grudniewska M, Jung I, Abegg D, Liu J, Childs-Disney JL, Gibaut QMR, Haniff HS, Adibekian A, Mouradian MM, Disney MD. Tong Y, et al. Proc Natl Acad Sci U S A. 2024 Jan 9;121(2):e2306682120. doi: 10.1073/pnas.2306682120. Epub 2024 Jan 5. Proc Natl Acad Sci U S A. 2024. PMID: 38181056
Fragment-Based Approaches to Identify RNA Binders.
Suresh BM, Taghavi A, Childs-Disney JL, Disney MD. Suresh BM, et al. J Med Chem. 2023 May 25;66(10):6523-6541. doi: 10.1021/acs.jmedchem.3c00034. Epub 2023 May 15. J Med Chem. 2023. PMID: 37186875 Review.
A blood-brain penetrant RNA-targeted small molecule triggers elimination of r(G4C2)exp in c9ALS/FTD via the nuclear RNA exosome.
Bush JA, Meyer SM, Fuerst R, Tong Y, Li Y, Benhamou RI, Aikawa H, Zanon PRA, Gibaut QMR, Angelbello AJ, Gendron TF, Zhang YJ, Petrucelli L, Heick Jensen T, Childs-Disney JL, Disney MD. Bush JA, et al. Proc Natl Acad Sci U S A. 2022 Nov 29;119(48):e2210532119. doi: 10.1073/pnas.2210532119. Epub 2022 Nov 21. Proc Natl Acad Sci U S A. 2022. PMID: 36409902 Free PMC article.
Ribonuclease recruitment using a small molecule reduced c9ALS/FTD r(G4C2) repeat expansion in vitro and in vivo ALS models.
Bush JA, Aikawa H, Fuerst R, Li Y, Ursu A, Meyer SM, Benhamou RI, Chen JL, Khan T, Wagner-Griffin S, Van Meter MJ, Tong Y, Olafson H, McKee KK, Childs-Disney JL, Gendron TF, Zhang Y, Coyne AN, Wang ET, Yildirim I, Wang KW, Petrucelli L, Rothstein JD, Disney MD. Bush JA, et al. Sci Transl Med. 2021 Oct 27;13(617):eabd5991. doi: 10.1126/scitranslmed.abd5991. Epub 2021 Oct 27. Sci Transl Med. 2021. PMID: 34705518 Free PMC article.
Small molecule recognition of disease-relevant RNA structures.
Meyer SM, Williams CC, Akahori Y, Tanaka T, Aikawa H, Tong Y, Childs-Disney JL, Disney MD. Meyer SM, et al. Chem Soc Rev. 2020 Oct 5;49(19):7167-7199. doi: 10.1039/d0cs00560f. Chem Soc Rev. 2020. PMID: 32975549 Free PMC article. Review.
38 results