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mRNA-based therapy in a rabbit model of variegate porphyria offers new insights into the pathogenesis of acute attacks.
Jericó D, Córdoba KM, Jiang L, Schmitt C, Morán M, Sampedro A, Alegre M, Collantes M, Santamaría E, Alegre E, Culerier C, de Mendoza AE, Oyarzabal J, Martín MA, Peñuelas I, Ávila MA, Gouya L, Martini PGV, Fontanellas A. Jericó D, et al. Among authors: jiang l. Mol Ther Nucleic Acids. 2021 May 19;25:207-219. doi: 10.1016/j.omtn.2021.05.010. eCollection 2021 Sep 3. Mol Ther Nucleic Acids. 2021. PMID: 34458006 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. Nat Med. 2018 Dec;24(12):1899-1909. doi: 10.1038/s41591-018-0199-z. Epub 2018 Oct 8. Nat Med. 2018. PMID: 30297912
Recombinant porphobilinogen deaminase targeted to the liver corrects enzymopenia in a mouse model of acute intermittent porphyria.
Córdoba KM, Serrano-Mendioroz I, Jericó D, Merino M, Jiang L, Sampedro A, Alegre M, Corrales F, Garrido MJ, Martini PGV, Lanciego JL, Prieto J, Berraondo P, Fontanellas A. Córdoba KM, et al. Among authors: jiang l. Sci Transl Med. 2022 Jan 12;14(627):eabc0700. doi: 10.1126/scitranslmed.abc0700. Epub 2022 Jan 12. Sci Transl Med. 2022. PMID: 35020410
Characterizing the mechanism of action for mRNA therapeutics for the treatment of propionic acidemia, methylmalonic acidemia, and phenylketonuria.
Baek R, Coughlan K, Jiang L, Liang M, Ci L, Singh H, Zhang H, Kaushal N, Rajlic IL, Van L, Dimen R, Cavedon A, Yin L, Rice L, Frassetto A, Guey L, Finn P, Martini PGV. Baek R, et al. Among authors: jiang l. Nat Commun. 2024 May 7;15(1):3804. doi: 10.1038/s41467-024-47460-9. Nat Commun. 2024. PMID: 38714648 Free PMC article.
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