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Page 1
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: lukacs c. 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.
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: lukacs c. 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.
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: lukacs cm. 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.
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: lukacs cm. Nat Commun. 2020 Oct 21;11(1):5339. doi: 10.1038/s41467-020-19156-3. Nat Commun. 2020. PMID: 33087718 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: lukacs cm. 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 as an etiological treatment for acute intermittent porphyria.
Jiang L, Berraondo P, Jericó D, Guey LT, Sampedro A, Frassetto A, Benenato KE, Burke K, Santamaría E, Alegre M, Pejenaute Á, Kalariya M, Butcher W, Park JS, Zhu X, Sabnis S, Kumarasinghe ES, Salerno T, Kenney M, Lukacs CM, Ávila MA, Martini PGV, Fontanellas A. Jiang L, et al. Among authors: lukacs cm. Nat Med. 2018 Dec;24(12):1899-1909. doi: 10.1038/s41591-018-0199-z. Epub 2018 Oct 8. Nat Med. 2018. PMID: 30297912
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: lukacs cm. 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.
44 results