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A deep learning approach to identify gene targets of a therapeutic for human splicing disorders.
Gao D, Morini E, Salani M, Krauson AJ, Chekuri A, Sharma N, Ragavendran A, Erdin S, Logan EM, Li W, Dakka A, Narasimhan J, Zhao X, Naryshkin N, Trotta CR, Effenberger KA, Woll MG, Gabbeta V, Karp G, Yu Y, Johnson G, Paquette WD, Cutting GR, Talkowski ME, Slaugenhaupt SA. Gao D, et al. Among authors: trotta cr. Nat Commun. 2021 Jun 7;12(1):3332. doi: 10.1038/s41467-021-23663-2. Nat Commun. 2021. PMID: 34099697 Free PMC article.
Small molecule splicing modifiers with systemic HTT-lowering activity.
Bhattacharyya A, Trotta CR, Narasimhan J, Wiedinger KJ, Li W, Effenberger KA, Woll MG, Jani MB, Risher N, Yeh S, Cheng Y, Sydorenko N, Moon YC, Karp GM, Weetall M, Dakka A, Gabbeta V, Naryshkin NA, Graci JD, Tripodi T Jr, Southwell A, Hayden M, Colacino JM, Peltz SW. Bhattacharyya A, et al. Among authors: trotta cr. Nat Commun. 2021 Dec 15;12(1):7299. doi: 10.1038/s41467-021-27157-z. Nat Commun. 2021. PMID: 34911927 Free PMC article.
PTC124 targets genetic disorders caused by nonsense mutations.
Welch EM, Barton ER, Zhuo J, Tomizawa Y, Friesen WJ, Trifillis P, Paushkin S, Patel M, Trotta CR, Hwang S, Wilde RG, Karp G, Takasugi J, Chen G, Jones S, Ren H, Moon YC, Corson D, Turpoff AA, Campbell JA, Conn MM, Khan A, Almstead NG, Hedrick J, Mollin A, Risher N, Weetall M, Yeh S, Branstrom AA, Colacino JM, Babiak J, Ju WD, Hirawat S, Northcutt VJ, Miller LL, Spatrick P, He F, Kawana M, Feng H, Jacobson A, Peltz SW, Sweeney HL. Welch EM, et al. Among authors: trotta cr. Nature. 2007 May 3;447(7140):87-91. doi: 10.1038/nature05756. Epub 2007 Apr 22. Nature. 2007. PMID: 17450125
The DHODH inhibitor PTC299 arrests SARS-CoV-2 replication and suppresses induction of inflammatory cytokines.
Luban J, Sattler RA, Mühlberger E, Graci JD, Cao L, Weetall M, Trotta C, Colacino JM, Bavari S, Strambio-De-Castillia C, Suder EL, Wang Y, Soloveva V, Cintron-Lue K, Naryshkin NA, Pykett M, Welch EM, O'Keefe K, Kong R, Goodwin E, Jacobson A, Paessler S, Peltz SW. Luban J, et al. Virus Res. 2021 Jan 15;292:198246. doi: 10.1016/j.virusres.2020.198246. Epub 2020 Nov 26. Virus Res. 2021. PMID: 33249060 Free PMC article.
Nonsense suppression activity of PTC124 (ataluren).
Peltz SW, Welch EM, Jacobson A, Trotta CR, Naryshkin N, Sweeney HL, Bedwell DM. Peltz SW, et al. Among authors: trotta cr. Proc Natl Acad Sci U S A. 2009 Jun 23;106(25):E64; author reply E65. doi: 10.1073/pnas.0901936106. Epub 2009 Jun 8. Proc Natl Acad Sci U S A. 2009. PMID: 19506240 Free PMC article. No abstract available.
Targeting post-transcriptional control for drug discovery.
Peltz SW, Welch EM, Trotta CR, Davis T, Jacobson A. Peltz SW, et al. Among authors: trotta cr. RNA Biol. 2009 Jul-Aug;6(3):329-34. doi: 10.4161/rna.6.3.8953. Epub 2009 Jul 7. RNA Biol. 2009. PMID: 19574739 Review.
The nucleoside analog clitocine is a potent and efficacious readthrough agent.
Friesen WJ, Trotta CR, Tomizawa Y, Zhuo J, Johnson B, Sierra J, Roy B, Weetall M, Hedrick J, Sheedy J, Takasugi J, Moon YC, Babu S, Baiazitov R, Leszyk JD, Davis TW, Colacino JM, Peltz SW, Welch EM. Friesen WJ, et al. Among authors: trotta cr. RNA. 2017 Apr;23(4):567-577. doi: 10.1261/rna.060236.116. Epub 2017 Jan 17. RNA. 2017. PMID: 28096517 Free PMC article.
Targeting of Hematologic Malignancies with PTC299, A Novel Potent Inhibitor of Dihydroorotate Dehydrogenase with Favorable Pharmaceutical Properties.
Cao L, Weetall M, Trotta C, Cintron K, Ma J, Kim MJ, Furia B, Romfo C, Graci JD, Li W, Du J, Sheedy J, Hedrick J, Risher N, Yeh S, Qi H, Arasu T, Hwang S, Lennox W, Kong R, Petruska J, Moon YC, Babiak J, Davis TW, Jacobson A, Almstead NG, Branstrom A, Colacino JM, Peltz SW. Cao L, et al. Mol Cancer Ther. 2019 Jan;18(1):3-16. doi: 10.1158/1535-7163.MCT-18-0863. Epub 2018 Oct 23. Mol Cancer Ther. 2019. PMID: 30352802 Free PMC article.
28 results