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Page 1
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: martini pgv. Nat Med. 2018 Dec;24(12):1899-1909. doi: 10.1038/s41591-018-0199-z. Epub 2018 Oct 8. Nat Med. 2018. PMID: 30297912
Messenger RNA therapy for rare genetic metabolic diseases.
Berraondo P, Martini PGV, Avila MA, Fontanellas A. Berraondo P, et al. Among authors: martini pgv. Gut. 2019 Jul;68(7):1323-1330. doi: 10.1136/gutjnl-2019-318269. Epub 2019 Feb 22. Gut. 2019. PMID: 30796097 Review.
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: martini pgv. 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.
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: martini pgv. 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: martini pgv. Nat Commun. 2024 May 7;15(1):3804. doi: 10.1038/s41467-024-47460-9. Nat Commun. 2024. PMID: 38714648 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: martini pgv. Nat Commun. 2020 Oct 21;11(1):5339. doi: 10.1038/s41467-020-19156-3. Nat Commun. 2020. PMID: 33087718 Free PMC article.
54 results