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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: zhuo j. 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 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: zhuo j. Nat Commun. 2021 May 25;12(1):3090. doi: 10.1038/s41467-021-23318-2. Nat Commun. 2021. PMID: 34035281 Free PMC article.
Dual mRNA therapy restores metabolic function in long-term studies in mice with propionic acidemia.
Jiang L, Park JS, Yin L, Laureano R, Jacquinet E, Yang J, Liang S, Frassetto A, Zhuo J, Yan X, Zhu X, Fortucci S, Hoar K, Mihai C, Tunkey C, Presnyak V, Benenato KE, Lukacs CM, Martini PGV, Guey LT. Jiang L, et al. Among authors: zhuo j. Nat Commun. 2020 Oct 21;11(1):5339. doi: 10.1038/s41467-020-19156-3. Nat Commun. 2020. PMID: 33087718 Free PMC article.
mRNA Therapy Improves Metabolic and Behavioral Abnormalities in a Murine Model of Citrin Deficiency.
Cao J, An D, Galduroz M, Zhuo J, Liang S, Eybye M, Frassetto A, Kuroda E, Funahashi A, Santana J, Mihai C, Benenato KE, Kumarasinghe ES, Sabnis S, Salerno T, Coughlan K, Miracco EJ, Levy B, Besin G, Schultz J, Lukacs C, Guey L, Finn P, Furukawa T, Giangrande PH, Saheki T, Martini PGV. Cao J, et al. Among authors: zhuo j. Mol Ther. 2019 Jul 3;27(7):1242-1251. doi: 10.1016/j.ymthe.2019.04.017. Epub 2019 Apr 23. Mol Ther. 2019. PMID: 31056400 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: zhuo j. 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.