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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. Among authors: meyer sm. 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.
Design of small molecules targeting RNA structure from sequence.
Ursu A, Childs-Disney JL, Andrews RJ, O'Leary CA, Meyer SM, Angelbello AJ, Moss WN, Disney MD. Ursu A, et al. Among authors: meyer sm. Chem Soc Rev. 2020 Oct 19;49(20):7252-7270. doi: 10.1039/d0cs00455c. Chem Soc Rev. 2020. PMID: 32935689 Free PMC article. Review.
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.
Reprogramming of Protein-Targeted Small-Molecule Medicines to RNA by Ribonuclease Recruitment.
Zhang P, Liu X, Abegg D, Tanaka T, Tong Y, Benhamou RI, Baisden J, Crynen G, Meyer SM, Cameron MD, Chatterjee AK, Adibekian A, Childs-Disney JL, Disney MD. Zhang P, et al. Among authors: meyer sm. J Am Chem Soc. 2021 Aug 25;143(33):13044-13055. doi: 10.1021/jacs.1c02248. Epub 2021 Aug 13. J Am Chem Soc. 2021. PMID: 34387474 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. Among authors: meyer sm. 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.
110 results