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mRNA therapy restores euglycemia and prevents liver tumors in murine model of glycogen storage disease.
Cao J, Choi M, Guadagnin E, Soty M, Silva M, Verzieux V, Weisser E, Markel A, Zhuo J, Liang S, Yin L, Frassetto A, Graham AR, Burke K, Ketova T, Mihai C, Zalinger Z, Levy B, Besin G, Wolfrom M, Tran B, Tunkey C, Owen E, Sarkis J, Dousis A, Presnyak V, Pepin C, Zheng W, Ci L, Hard M, Miracco E, Rice L, Nguyen V, Zimmer M, Rajarajacholan U, Finn PF, Mithieux G, Rajas F, Martini PGV, Giangrande PH. Cao J, et al. Among authors: presnyak v. Nat Commun. 2021 May 25;12(1):3090. doi: 10.1038/s41467-021-23318-2. Nat Commun. 2021. PMID: 34035281 Free PMC article.
Synthetic human ABCB4 mRNA therapy rescues severe liver disease phenotype in a BALB/c.Abcb4-/- mouse model of PFIC3.
Wei G, Cao J, Huang P, An P, Badlani D, Vaid KA, Zhao S, Wang DQ, Zhuo J, Yin L, Frassetto A, Markel A, Presnyak V, Gandham S, Hua S, Lukacs C, Finn PF, Giangrande PH, Martini PGV, Popov YV. Wei G, et al. Among authors: presnyak v. J Hepatol. 2021 Jun;74(6):1416-1428. doi: 10.1016/j.jhep.2020.12.010. Epub 2020 Dec 17. J Hepatol. 2021. PMID: 33340584 Free PMC article.
mRNA structure regulates protein expression through changes in functional half-life.
Mauger DM, Cabral BJ, Presnyak V, Su SV, Reid DW, Goodman B, Link K, Khatwani N, Reynders J, Moore MJ, McFadyen IJ. Mauger DM, et al. Among authors: presnyak v. Proc Natl Acad Sci U S A. 2019 Nov 26;116(48):24075-24083. doi: 10.1073/pnas.1908052116. Epub 2019 Nov 11. Proc Natl Acad Sci U S A. 2019. PMID: 31712433 Free PMC article.
Systemic mRNA Therapy for the Treatment of Fabry Disease: Preclinical Studies in Wild-Type Mice, Fabry Mouse Model, and Wild-Type Non-human Primates.
Zhu X, Yin L, Theisen M, Zhuo J, Siddiqui S, Levy B, Presnyak V, Frassetto A, Milton J, Salerno T, Benenato KE, Milano J, Lynn A, Sabnis S, Burke K, Besin G, Lukacs CM, Guey LT, Finn PF, Martini PGV. Zhu X, et al. Among authors: presnyak v. Am J Hum Genet. 2019 Apr 4;104(4):625-637. doi: 10.1016/j.ajhg.2019.02.003. Epub 2019 Mar 14. Am J Hum Genet. 2019. PMID: 30879639 Free PMC article.
Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia.
An D, Schneller JL, Frassetto A, Liang S, Zhu X, Park JS, Theisen M, Hong SJ, Zhou J, Rajendran R, Levy B, Howell R, Besin G, Presnyak V, Sabnis S, Murphy-Benenato KE, Kumarasinghe ES, Salerno T, Mihai C, Lukacs CM, Chandler RJ, Guey LT, Venditti CP, Martini PGV. An D, et al. Among authors: presnyak v. Cell Rep. 2017 Dec 19;21(12):3548-3558. doi: 10.1016/j.celrep.2017.11.081. Cell Rep. 2017. PMID: 29262333 Free PMC article.
Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia.
An D, Schneller JL, Frassetto A, Liang S, Zhu X, Park JS, Theisen M, Hong SJ, Zhou J, Rajendran R, Levy B, Howell R, Besin G, Presnyak V, Sabnis S, Murphy-Benenato KE, Kumarasinghe ES, Salerno T, Mihai C, Lukacs CM, Chandler RJ, Guey LT, Venditti CP, Martini PGV. An D, et al. Among authors: presnyak v. Cell Rep. 2018 Aug 28;24(9):2520. doi: 10.1016/j.celrep.2018.08.049. Cell Rep. 2018. PMID: 30157442 Free article. No abstract available.
23 results